A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations
(University of Alabama at Birmingham) An experimental drug reported in Nature Communications suggests that a " path is clearly achievable " to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic fibrosis patients, as well as patients with other genetic diseases, including Duchenne muscular dystrophy,β-thalassemia and numerous types of cancers, that are also caused by nonsense mutations. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 19, 2021 Category: International Medicine & Public Health Source Type: news

Roche ’s ENSPRYNG approved by European Commission as first and only at-home subcutaneous treatment for neuromyelitis optica spectrum disorder (NMOSD)
Basel, 28 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved ENSPRYNG ® (satralizumab) for the treatment of adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). ENSPRYNG is the first and only NMO SD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training.“An NMOSD relapse can be devastating, causing permanent neurological damage ...
Source: Roche Investor Update - June 28, 2021 Category: Pharmaceuticals Source Type: news

Roche ’s ENSPRYNG approved by European Commission as first and only at-home subcutaneous treatment for neuromyelitis optica spectrum disorder (NMOSD)
Basel, 28 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved ENSPRYNG ® (satralizumab) for the treatment of adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). ENSPRYNG is the first and only NMO SD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training.“An NMOSD relapse can be devastating, causing permanent neurological damage ...
Source: Roche Media News - June 28, 2021 Category: Pharmaceuticals Source Type: news

What Are the Stages of Muscular Dystrophy?
Title: What Are the Stages of Muscular Dystrophy?Category: Diseases and ConditionsCreated: 6/17/2021 12:00:00 AMLast Editorial Review: 6/17/2021 12:00:00 AM (Source: MedicineNet Kids Health General)
Source: MedicineNet Kids Health General - June 17, 2021 Category: Pediatrics Source Type: news

A Space in Time review – lyrical portrait of a family facing an incurable disorder
This poignant documentary about two young brothers with Duchenne muscular dystrophy celebrates the power of love and togethernessHere is a deeply personal documentary that raises awareness about a disability without neglecting the interiority of those living with the condition. Co-directed by Riccardo Servini and Nick Taussig, the film follows the Taussig family ’s experience of Duchenne muscular dystrophy, a genetic disorder that affects their young sons, Theo and Oscar.Duchenne is incurable and fatal. By the time Theo and Oscar are in their teens, they will be using wheelchairs full time; their 20s will come with v...
Source: Guardian Unlimited Science - May 10, 2021 Category: Science Authors: Phuong Le Tags: Film Documentary films Health Society Culture Genetics Science Children Parents and parenting Family Biology Life and style Source Type: news

Mother and son share rare muscle disorder, treated decades apart by same doctor
Twenty-four years ago, Bobbi Heinold, 17, was diagnosed with facioscapulohumeral muscular dystrophy, a genetic muscle condition characterized by weakness, especially in the face, shoulder blades and upper arms. Doctors told her it could affect her quality of life. Bobbi and her mom drove from their Indiana home to Mayo Clinic in Rochester, Minnesota, to learn [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - May 6, 2021 Category: Databases & Libraries Source Type: news

A milestone in muscular dystrophy therapy
(Max Delbr ü ck Center for Molecular Medicine in the Helmholtz Association) A new gene-editing technique can be used to correct mutations in muscle stem cells, paving the way for the first potential cell therapy for genetic muscle disorders. The ECRC team led by Professor Simone Spuler have published their findings in the journal JCI Insight. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 30, 2021 Category: International Medicine & Public Health Source Type: news

CHMP recommends EU approval of Roche ’s ENSPRYNG (satralizumab) for adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD)
Basel, 23 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency ’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ENSPRYNG® (satralizumab) as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spe ctrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). AQP4-IgG are present in around 70-80% of people with NMOSD, who tend to experience a more severe disease course.NMOS...
Source: Roche Media News - April 23, 2021 Category: Pharmaceuticals Source Type: news

CHMP recommends EU approval of Roche ’s ENSPRYNG (satralizumab) for adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD)
Basel, 23 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency ’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ENSPRYNG® (satralizumab) as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spe ctrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). AQP4-IgG are present in around 70-80% of people with NMOSD, who tend to experience a more severe disease course.NMOS...
Source: Roche Investor Update - April 23, 2021 Category: Pharmaceuticals Source Type: news

C-Path opens access to Duchenne Regulatory Science Consortium database
(Critical Path Institute (C-Path)) C-Path announced today that it will open access to the Duchenne Regulatory Science Consortium database to qualified researchers, through its Rare Disease Cures Accelerator, Data and Analytics Platform. The D-RSC database includes data from Duchenne muscular dystrophy clinical trials, natural history studies and clinical data collections. The contributors of these datasets have given permission for this data to be shared externally to accelerate therapy development for DMD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 22, 2021 Category: International Medicine & Public Health Source Type: news

New data for Roche ’s OCREVUS (ocrelizumab) reinforce significant benefit on slowing disease progression in relapsing and primary progressive multiple sclerosis
Basel, 16 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new OCREVUS ® (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. These data are being presented virtually at the 73rd American Academy of Neurology (AAN) Annual Meeting from 17–22 April 2021. OCREVUS is the number one prescribed MS medication in the U.S. for patients starting a new treatment, and more than 200,000 peop...
Source: Roche Investor Update - April 16, 2021 Category: Pharmaceuticals Source Type: news

New data for Roche ’s OCREVUS (ocrelizumab) reinforce significant benefit on slowing disease progression in relapsing and primary progressive multiple sclerosis
Basel, 16 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new OCREVUS ® (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. These data are being presented virtually at the 73rd American Academy of Neurology (AAN) Annual Meeting from 17–22 April 2021. OCREVUS is the number one prescribed MS medication in the U.S. for patients starting a new treatment, and more than 200,000 peop...
Source: Roche Media News - April 16, 2021 Category: Pharmaceuticals Source Type: news

Novel muscular dystrophy gene connects to a key biological pathway
(University of Minnesota Medical School) New research from the University of Minnesota Medical School found mutations in a novel gene that may help identify patients with a specific form of muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 15, 2021 Category: International Medicine & Public Health Source Type: news

New discovery could lead to therapies for patients with Duchenne muscular dystrophy
(University of California - Irvine) A new study, led by the University of California, Irvine (UCI), reveals how chronic inflammation promotes muscle fibrosis, which could inform the development of new therapies for patients suffering from Duchenne muscular dystrophy (DMD), a fatal muscle disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 14, 2021 Category: International Medicine & Public Health Source Type: news

Biotech company spun out of Ohio State University research raises $116M in latest round
A biotechnology company that spun out of Ohio State University research announced it raised more than $116 million in its latest financing round. Boston-based Entrada Therapeutics Inc., which was co-founded by an OSU professor, said the investment will support treating people with multiple neuromuscular diseases, including Duchenne muscular dystrophy. When it was founded in 2018, Entrada raised $59 million, which was the largest amount rai sed in an initial financing round for an OSU startup. The… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 5, 2021 Category: Biotechnology Authors: Hayleigh Colombo Source Type: news

Chemical cocktail creates new avenues for muscle stem cell therapies
A UCLA-led research team has identified a chemical cocktail that enables the production of large numbers of muscle stem cells, which can self-renew and give rise to all types of skeletal muscle cells.The advance could lead to the development of stem cell-based therapies for muscle loss or damage due to injury, age or disease.The research was published in Nature Biomedical Engineering.Muscle stem cells are responsible for muscle growth, repair and regeneration following injury throughout a person ’s life. In fully grown adults, muscle stem cells are quiescent — they remain inactive until they are called to respo...
Source: UCLA Newsroom: Health Sciences - March 18, 2021 Category: Universities & Medical Training Source Type: news

New two-year data show Roche ’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
Basel, 16 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi ™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. Th...
Source: Roche Investor Update - March 16, 2021 Category: Pharmaceuticals Source Type: news

New two-year data show Roche ’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
Basel, 16 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi ™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. Th...
Source: Roche Media News - March 16, 2021 Category: Pharmaceuticals Source Type: news

Translating passion into execution: Cure Rare Disease's founder on the business of biotech
When a career path chooses you in the form of a pressing personal cause, passion and commitment must soon be followed by execution. And that means schooling up — quickly. I had heard the call of entrepreneurship early. I had a new Harvard Business School degree in hand at the same time my brother’s health was declining steeply due to Duchenne muscular dystrophy. His fatal degenerative neuromuscular condition wouldn't wait, and the lack of viable option s in the pipeline for his gene mutation required entrepreneurial thinking. The science would come. I was in Boston, after all, immersed in bioscience. And it has...
Source: bizjournals.com Health Care:Biotechnology headlines - February 26, 2021 Category: Biotechnology Authors: Rich Horgan Source Type: news

FDA OKs First'Targeted Treatment' for Rare DMD Mutation FDA OKs First'Targeted Treatment' for Rare DMD Mutation
The FDA has approved casimersen (Amondys 45) injection for treating patients with both Duchenne muscular dystrophy (DMD) and a rare mutation of the DMD gene.FDA Approvals (Source: Medscape Pediatrics Headlines)
Source: Medscape Pediatrics Headlines - February 26, 2021 Category: Pediatrics Tags: Neurology & Neurosurgery News Alert Source Type: news

FDA Approves Amondys 45 (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Amondys 45... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - February 25, 2021 Category: Drugs & Pharmacology Source Type: news

For UCLA-based startup, new muscular dystrophy treatment is a personal mission
Courtney Young helped develop the gene therapy at the heart of a biotech startup,MyoGene Bio, when she was a doctoral student at UCLA from 2013 to 2018. But the kernel of the company ’s endeavor is older still — and definitively personal.In 2008, when Young was in high school, she found out that her cousin, then just a toddler, had been diagnosed with Duchenne muscular dystrophy. She made it her mission to cultivate the skills needed to provide better options for him and others who were suffering with the deadly muscle-wasting disease.“I directed my career toward working on Duchenne,” said Young, My...
Source: UCLA Newsroom: Health Sciences - February 23, 2021 Category: Universities & Medical Training Source Type: news

After the Nobel, what next for Crispr  gene-editing therapies?
Hailed as the ‘molecular scissors’ that will allow us to rewrite our genes, the DNA tool is being trialled in treatments for everything from sickle-cell anaemia to cancerWhen last year ’s Nobel prize for chemistry was awarded tobiochemist Jennifer Doudna and microbiologist Emmanuelle Charpentier for their work in developing the technique of gene editing known as Crispr-Cas9 (pronounced “crisper”), headlines hailed their discovery as “molecular scissors” that would allow us to “rewrite the book of life” – with all the complicated ethical questions that ability rais...
Source: Guardian Unlimited Science - February 21, 2021 Category: Science Authors: Philip Ball Tags: Gene editing Medical research Genetics Biology Science Cancer Huntington's disease Sickle cell disease Source Type: news

Invitation to Roche ’s virtual event on 24-month data of Evrysdi in patients with type 2 or type 3 spinal muscular atrophy presented at the 2021 MDA virtual conference
We are pleased to invite investors and analysts to participate in our virtual event on Friday, 19 March, 2021, highlighting Roche data presented during the 2021 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 15th to 18th February. (Source: Roche Investor Update)
Source: Roche Investor Update - February 19, 2021 Category: Pharmaceuticals Source Type: news

The Hardship of Social Distancing When Touch Is a Lifeline
For me, and many others with physical disabilities, touch is not merely a luxury or a pleasure, but an aspect of my functionality, my basic survival. (Source: NYT Health)
Source: NYT Health - February 10, 2021 Category: Consumer Health News Authors: Sonali Gupta Tags: Touch (Sense) Muscular Dystrophy Quarantines Muscles Genetics and Heredity Coronavirus (2019-nCoV) Physical Therapy Disabilities Source Type: news

Early Promise for Therapy Against Duchenne Muscular Dystrophy
THURSDAY, Jan. 28, 2021 -- An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests. The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States --... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 28, 2021 Category: General Medicine Source Type: news

What Is the Best Treatment for Muscular Dystrophy?
Title: What Is the Best Treatment for Muscular Dystrophy?Category: Diseases and ConditionsCreated: 1/28/2021 12:00:00 AMLast Editorial Review: 1/28/2021 12:00:00 AM (Source: MedicineNet Kids Health General)
Source: MedicineNet Kids Health General - January 28, 2021 Category: Pediatrics Source Type: news

Enhanced oral uptake of exosomes opens cell therapy alternative
(Cedars-Sinai Medical Center) Cell-derived exosomes are effective in treating disease when mixed with the dominant protein in breast milk and given orally, a new Smidt Heart Institute study of laboratory mice shows. The findings, published in the peer-reviewed Journal of Extracellular Vesicles, could help develop new oral medications for treating patients with muscular dystrophy and heart failure. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 12, 2021 Category: International Medicine & Public Health Source Type: news

Science Saturday: Across time, place to find cures for inherited heart diseases
Five years ago, Jay Schneider, M.D., Ph.D., was running a lab at The University of Texas Southwestern Medical Center in Dallas. The lab focused on researching cures for the genetic muscular-wasting disorder, Duchenne muscular dystrophy. His wife, Alice Chang, M.D., had accepted an offer to work at Mayo Clinic in Rochester, Minnesota, two years before. The [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - January 2, 2021 Category: Databases & Libraries Source Type: news

Digging deep for differences in Duchenne muscular dystrophy
(UT Southwestern Medical Center) A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 21, 2020 Category: International Medicine & Public Health Source Type: news

FDA approves Roche ’s OCREVUS® (ocrelizumab) shorter 2-hour infusion for relapsing and primary progressive multiple sclerosis
Basel, 14 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a shorter two-hour infusion time for OCREVUS ® (ocrelizumab), dosed twice-yearly for those living with relapsing or primary progressive multiple sclerosis (MS) who have not experienced any prior serious infusion reactions (IRs). The approval was based on data from the randomised, double-blind ENSEMBLE PLUS study.“More than 170,000 people with MS have been treated with OCREVUS - the only approved B-cell therapy with a twice-yearly dosing schedule - and it is the most prescribed...
Source: Roche Investor Update - December 14, 2020 Category: Pharmaceuticals Source Type: news

FDA approves Roche ’s OCREVUS® (ocrelizumab) shorter 2-hour infusion for relapsing and primary progressive multiple sclerosis
Basel, 14 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a shorter two-hour infusion time for OCREVUS ® (ocrelizumab), dosed twice-yearly for those living with relapsing or primary progressive multiple sclerosis (MS) who have not experienced any prior serious infusion reactions (IRs). The approval was based on data from the randomised, double-blind ENSEMBLE PLUS study.“More than 170,000 people with MS have been treated with OCREVUS - the only approved B-cell therapy with a twice-yearly dosing schedule - and it is the most prescribed...
Source: Roche Media News - December 14, 2020 Category: Pharmaceuticals Source Type: news

Muscle cell secrets
(Max Delbr ü ck Center for Molecular Medicine in the Helmholtz Association) A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are. The study, which has been published in Nature Communications, can help us better understand muscle diseases such as Duchenne muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 11, 2020 Category: International Medicine & Public Health Source Type: news

PTC Announces Translarna(TM) Approval in Russia for the Treatment of Duchenne Muscular Dystrophy
SOUTH PLAINFIELD, N.J., Dec. 4, 2020 -- (Healthcare Sales & Marketing Network) -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenn... Biopharmaceuticals, Regulatory PTC Therapeutics, Translarna, ataluren, Duchenne muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 4, 2020 Category: Pharmaceuticals Source Type: news

How Long Can You Live with Muscular Dystrophy
? (Source: eMedicineHealth.com)
Source: eMedicineHealth.com - November 5, 2020 Category: General Medicine Source Type: news

Genetic mutation could worsen heart function in Duchenne muscular dystrophy patients
(UT Southwestern Medical Center) DALLAS - Nov. 4, 2020 - A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in theJournal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients' lives. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 4, 2020 Category: International Medicine & Public Health Source Type: news

Ataluren Delays Disease Milestones in Nonsense Mutation DMD Ataluren Delays Disease Milestones in Nonsense Mutation DMD
Long-term treatment with ataluren delays loss of ambulation and may delay decline in pulmonary function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD), according to study results.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - October 28, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

26.10.20: Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapy
Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapyAcquisition fuels Bayer's cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading AAV-based gene therapy platform already yielding commercial and clinical stage assets with potential of helping larger patient populations / Portfolio includes investigational pre-clinical and clinical stage development candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases such as ...
Source: Bayer IR Newsfeed: Events - October 26, 2020 Category: Pharmaceuticals Source Type: news

Bayer-acquires-Asklepios-BioPharmaceutical-to-broaden-innovation-base-in-cell-and-gene-therapy
Acquisition fuels Bayer’s cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading AAV-based gene therapy platform already yielding commercial and clinical stage assets with potential of helping larger patient populations / Portfolio includes investigational pre-clinical and clinical stage development candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases such as therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure, ...
Source: Bayer Company News - October 26, 2020 Category: Pharmaceuticals Source Type: news

Scientists identify compound that stimulates muscle cells in mice
UCLA researchers have identified a compound that can reproduce the effect of exercise in muscle cells in mice. Thefindings are published in the journal Cell Reports Medicine.Normally, muscles get stronger as they are used, thanks to a series of chemical signals inside muscle cells. The newly identified compound activates those signals, which suggests that compounds like it could eventually be used to treat people with limb girdle muscular dystrophy, a form of adolescent-onset muscular dystrophy.When muscles aren ’t worked regularly, they gradually atrophy. (The phenomenon is familiar to anyone who’s had a cast ...
Source: UCLA Newsroom: Health Sciences - October 21, 2020 Category: Universities & Medical Training Source Type: news

Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001, its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Dear Investor, Please find attached a press release from our partner Sarepta Therapeutics:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-reports-sustained-functional-improvement Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - September 28, 2020 Category: Pharmaceuticals Source Type: news

New Duchenne muscular dystrophy drug shows benefit in Duke trial
(Duke University Medical Center) A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 25, 2020 Category: International Medicine & Public Health Source Type: news

Scientists uncover a novel approach to treating Duchenne muscular dystrophy
(Sanford Burnham Prebys Medical Discovery Institute) Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Universit à Cattolica del Sacro Cuore have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition, and has far-reaching implications for the field of regenerative medicine. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - September 15, 2020 Category: Biology Source Type: news

Drugging the undruggable: Yale finds treatment path for muscular dystrophy
Researchers have identified a possible treatment for Duchenne muscular dystrophy, a rare genetic disease for which there is currently no cure or treatment. (Source: Yale Science and Health News)
Source: Yale Science and Health News - September 11, 2020 Category: Universities & Medical Training Source Type: news

New data further reinforce Roche ’s OCREVUS (ocrelizumab) as a highly effective treatment for people with multiple sclerosis
Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). Subgroup analys is from the two-year open-label Phase IIIb CASTING study also demonstrates that patients benefit across a wide range of disease related and demographic subgroups, regardless of prior treatment background. Findings will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee...
Source: Roche Media News - September 11, 2020 Category: Pharmaceuticals Source Type: news

New data further reinforce Roche ’s OCREVUS (ocrelizumab) as a highly effective treatment for people with multiple sclerosis
Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). Subgroup analys is from the two-year open-label Phase IIIb CASTING study also demonstrates that patients benefit across a wide range of disease related and demographic subgroups, regardless of prior treatment background. Findings will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee...
Source: Roche Investor Update - September 11, 2020 Category: Pharmaceuticals Source Type: news

Drugging the undruggable: Yale finds treatment path for muscular dystrophy
(Yale University) Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered " undruggable. " The finding appears in the Aug. 25 edition of Science Signaling. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 11, 2020 Category: International Medicine & Public Health Source Type: news

New data show Roche ’s ENSPRYNG (satralizumab) significantly reduces severity and risk of relapse in neuromyelitis optica spectrum disorder (NMOSD)
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity ...
Source: Roche Investor Update - September 10, 2020 Category: Pharmaceuticals Source Type: news

New data show Roche ’s ENSPRYNG (satralizumab) significantly reduces severity and risk of relapse in neuromyelitis optica spectrum disorder (NMOSD)
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity ...
Source: Roche Media News - September 10, 2020 Category: Pharmaceuticals Source Type: news

Roche expands its multiple sclerosis portfolio with investigational BTK inhibitor fenebrutinib and initiates novel clinical trials for OCREVUS (ocrelizumab)
Basel, 9 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the initiation of an innovative Phase III clinical trial programme for its investigational medicine fenebrutinib in multiple sclerosis (MS), along with a higher-dose Phase III clinical trial programme for OCREVUS® (ocrelizumab) and a distinct O CREVUS trial specifically to support African-American and Hispanic- and Latinx-American patients with MS. Overviews of clinical trials and scientific rationale will be presented at MSVirtual2020, the 8th Joint Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTR...
Source: Roche Investor Update - September 9, 2020 Category: Pharmaceuticals Source Type: news