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 Media News - 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 Investor Update - 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 Media News - 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 Investor Update - 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 Media News - September 9, 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

Treatments for Inherited Neuromuscular Diseases of Childhood Treatments for Inherited Neuromuscular Diseases of Childhood
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 27, 2020 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

Duchenne: " Crosstalk " between muscle and spleen
(University of Bonn) Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before the third decade of life. Researchers of the Universities of Maynooth (Ireland) and Bonn have found a connection between dystrophic muscles and the lymphatic system in mice with Duchenne disease. The results have now been published in the journal " iScience " . (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 27, 2020 Category: International Medicine & Public Health Source Type: news

Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - August 25, 2020 Category: Drugs & Pharmacology Source Type: news

Drug Trials Snapshots: VILTEPSO
VILTEPSO is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - August 18, 2020 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Viltepso (viltolarsen) Injection Granted Accelerated Approval for Rare Duchenne Muscular Dystrophy Mutation
THURSDAY, Aug. 13, 2020 -- Accelerated approval has been granted to Viltepso (viltolarsen) injection for treatment of patients with Duchenne muscular dystrophy (DMD) and a mutation of the DMD gene amenable to exon 53 skipping, the U.S. Food and Drug... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - August 13, 2020 Category: General Medicine Source Type: news

FDA OKs Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy FDA OKs Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
Viltolarsen is for patients with Duchenne muscular dystrophy with a confirmed mutation amenable to exon 53 skipping.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 13, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

FDA Approves Viltepso (viltolarsen) for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy
PARAMUS, N.J., Aug. 12, 2020 /PRNewswire/ -- NS Pharma, Inc. announced today that the U.S. Food& Drug Administration (FDA) has approved Viltepso (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 12, 2020 Category: Drugs & Pharmacology Source Type: news

A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived
(Source: NPR Health and Science)
Source: NPR Health and Science - July 27, 2020 Category: Consumer Health News Authors: Jon Hamilton Source Type: news

Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
Dear Investor, Please find attached a press release by Sarepta Therapeutics:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-receives-fast-track-designation-srp-9001 Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - July 24, 2020 Category: Pharmaceuticals Source Type: news

Researchers Hope Experimental Gene Therapy Is An Answer To A Fatal Genetic Disorder
Researchers believe gene therapy is poised to change the lives of thousands with the fatal genetic disorder Duchenne muscular dystrophy — thanks to over 40 years of scientist Jude Samulski's work. (Source: NPR Health and Science)
Source: NPR Health and Science - July 20, 2020 Category: Consumer Health News Authors: Jon Hamilton Source Type: news

Hansa Biopharma Announces Exclusive Agreement With Sarepta Therapeutics to Develop and Promote Imlifidase as Pre-treatment Ahead of Gene Therapy in Select Indications
Hansa grants Sarepta exclusive license to develop and promote imlifidase as a potential pre-treatment prior to the administration of gene therapy in Duchenne muscular dystrophy and Limb-girdle muscular dystrophy, for patients with neutralizing antibodies (... Biopharmaceuticals, Licensing Hansa Biopharma, Sarepta Therapeutics, imlifidase, muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 2, 2020 Category: Pharmaceuticals Source Type: news

New treatment for common form of muscular dystrophy shows promise in cells, animals
(University of Alberta Faculty of Medicine& Dentistry) Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences. Toshifumi Yokota, professor of medical genetics at the University of Alberta, led a team from Canada and the U.S. to create and test synthetic DNA-like molecules that interfere with the production of a toxic protein that destroys the muscles of people who have facioscapulohumeral muscular dystrophy (FSHD). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 29, 2020 Category: International Medicine & Public Health Source Type: news

Facioscapulohumeral muscular dystrophy community speaks to the FDA
FSHD Society holds externally led Patient-Focused Drug Development Meeting on FSH muscular dystrophy(PRWeb June 29, 2020)Read the full story at https://www.prweb.com/releases/facioscapulohumeral_muscular_dystrophy_community_speaks_to_the_fda/prweb17220588.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 29, 2020 Category: Pharmaceuticals Source Type: news

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the Company has completed the submission of a rolling New Drug Application... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - June 26, 2020 Category: Drugs & Pharmacology Source Type: news

New structural 'map' solves mysteries of gigantic gene regulator
(Walter and Eliza Hall Institute) Structural biology has been used to 'map' part of a protein called SMCHD1, explaining how some changes in SMCHD1 cause certain developmental and degenerative conditions.Publishing in the journal Science Signaling, the Walter and Eliza Hall Institute team revealed the structure of the portion of the SMCHD1 protein that is crucial to its function in 'switching off' genes. Inherited mutations in this part of SMCHD1 have been linked to a developmental disorder and a form of muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 17, 2020 Category: International Medicine & Public Health Source Type: news

Crystal structure of the hinge domain of Smchd1 reveals its dimerization mode and nucleic acid-binding residues
Structural maintenance of chromosomes flexible hinge domain containing 1 (SMCHD1) is an epigenetic regulator in which polymorphisms cause the human developmental disorder, Bosma arhinia micropthalmia syndrome, and the degenerative disease, facioscapulohumeral muscular dystrophy. SMCHD1 is considered a noncanonical SMC family member because its hinge domain is C-terminal, because it homodimerizes rather than heterodimerizes, and because SMCHD1 contains a GHKL-type, rather than an ABC-type ATPase domain at its N terminus. The hinge domain has been previously implicated in chromatin association; however, the underlying mechan...
Source: Signal Transduction Knowledge Environment - June 15, 2020 Category: Science Authors: Chen, K., Birkinshaw, R. W., Gurzau, A. D., Wanigasuriya, I., Wang, R., Iminitoff, M., Sandow, J. J., Young, S. N., Hennessy, P. J., Willson, T. A., Heckmann, D. A., Webb, A. I., Blewitt, M. E., Czabotar, P. E., Murphy, J. M. Tags: STKE Research Articles Source Type: news

First report of systemic delivery of micro-dystrophin gene therapy in children with DMD
(Nationwide Children's Hospital) Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) -- and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 15, 2020 Category: International Medicine & Public Health Source Type: news

UT Southwestern, Children's Health recognized for care of Duchenne muscular dystrophy
(UT Southwestern Medical Center) A joint program of UT Southwestern Medical Center and Children's Health has been approved as a Certified Duchenne Care Center (CDCC) by Parent Project Muscular Dystrophy (PPMD), the nation's most comprehensive nonprofit organization focused on finding a cure for Duchenne muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 27, 2020 Category: International Medicine & Public Health Source Type: news

Care-giver burden substantial in Duchenne muscular dystrophy in Spain
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - April 30, 2020 Category: Drugs & Pharmacology Source Type: news

Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p
Source: Roche Media News - April 28, 2020 Category: Pharmaceuticals Source Type: news

Substituting the next-best protein
(University of Ottawa) Children born with Duchenne muscular dystrophy have a mutation in the X-chromosome gene that would normally code for dystrophin, a protein that provides structural integrity to skeletal muscles. The loss of this protein causes severe symptoms, including deteriorating muscle strength beginning around the age of four. While there is no cure, a promising area of research has developed around the protein utrophin, which is ~ 80% identical to dystrophin and even takes its place early during muscle development. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 24, 2020 Category: International Medicine & Public Health Source Type: news

Study finds that lithium could be used to treat people with a rare muscle disease
(Natural News) A rare disease called limb-girdle muscular dystrophy D1 (LMGD1) makes even everyday actions such as climbing stairs, lifting objects and standing up from a chair difficult to do. Over time, it can even cause some people to lose the ability to walk. A recent study, however, shows that lithium can be a possible treatment for LMGD1. Researchers... (Source: NaturalNews.com)
Source: NaturalNews.com - March 30, 2020 Category: Consumer Health News Source Type: news

New treatment to slow muscle wastage in boys with DMD
A medicine developed by EU-funded researchers has been approved to treat children with the degenerative and fatal genetic disease Duchenne muscular dystrophy. A major clinical trial is expected to announce positive results soon. (Source: EUROPA - Research Information Centre)
Source: EUROPA - Research Information Centre - March 19, 2020 Category: Research Source Type: news

Releasing brakes: Potential new methods for Duchenne muscular dystrophy therapies
(University of Pennsylvania School of Medicine) Testing of small molecules in mouse models for Duchenne muscular dystrophy shows promise for restoration of muscle structure and function. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 24, 2020 Category: International Medicine & Public Health Source Type: news

Avidity Biosciences Appoints Joseph Baroldi as Chief Operating Officer
SAN DIEGO, Feb. 12, 2020 -- (Healthcare Sales & Marketing Network) -- Avidity Biosciences, Inc. (Avidity), a privately-held biotechnology company pioneering Antibody Oligonucleotide Conjugates (AOCs™), announced today the appointment of Joseph Baroldi as ... Biopharmaceuticals, Personnel Avidity Biosciences, Antibody-Oligonucleotide Conjugates, muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 12, 2020 Category: Pharmaceuticals Source Type: news