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 16, 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 - May 1, 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

'I trust you': How a Lancaster family helped lay the building blocks for gene-therapy breakthroughs at Nationwide Children ’s
Andrew is the “And” in Andelyn Biosciences. He was 3 in 2001 when he was diagnosed with Duchenne muscular dystrophy, the most common and among the most aggressive forms of the inherited condition. Patients usually don’t reach 20. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 30, 2020 Category: Biotechnology Authors: Carrie Ghose Source Type: news

Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,&rdqu...
Source: UCLA Newsroom: Health Sciences - January 28, 2020 Category: Universities & Medical Training Source Type: news

Gene scissors against incurable muscular disease
(Technical University of Munich (TUM)) Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum M ü nchen) have developed a gene therapy that may provide permanent relief for those suffering from DMD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 27, 2020 Category: International Medicine & Public Health Source Type: news

Heart-function protein may help muscular dystrophy patients live longer
(Rutgers University) Rutgers-led discovery may help prevent muscular dystrophy-related heart disease, the leading cause of death in patients with Duchenne muscular dystrophy (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 14, 2020 Category: Biology Source Type: news

The impact of gun violence on those already dying: perspectives from a palliative care physician - Vente TM.
The consult was for a 19-year-old man with Duchenne muscular dystrophy who was in the ICU with respiratory distress and weight loss. Given his degenerative disease, establishing care with the palliative care team made perfect sense. It was, by all mean... (Source: SafetyLit)
Source: SafetyLit - January 8, 2020 Category: International Medicine & Public Health Tags: Commentary Source Type: news

Biotech firm makes $2.8B deal to license muscular dystrophy gene therapy
A gene therapy invented at Nationwide Children's Hospital just landed a licensing deal worth as much as $2.8 billion for a Boston biotech, and now the company's Columbus, Ohio office will play a big role in getting the treatment to the market. Sarepta Therapeutics Inc. has licensed the commercial rights outside of the United States to the Swiss pharmaceutical giant Roche for $1.1 billion in cash and stock up front. Sarepta also can earn as much as $1.7 billion in milestone payments, plus royalties.… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 30, 2019 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news

Drug Trials Snapshots: VYONDYS 53
VYONDYS 53 is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 26, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Roche Dives Deeper Into Gene Therapy With $1.15 Bln Sarepta Licensing Deal Roche Dives Deeper Into Gene Therapy With $1.15 Bln Sarepta Licensing Deal
Roche Holding AG made its second major gene therapy deal in a year on Monday, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc's investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States.Reuters Health Information (Source: Medscape Critical Care Headlines)
Source: Medscape Critical Care Headlines - December 24, 2019 Category: Intensive Care Tags: Family Medicine/Primary Care News Source Type: news

Roche dives deeper into gene therapy with $1.15 billion Sarepta licensing deal
Roche Holding AG made its second major gene therapy deal in a year on Monday, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc's investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States. (Source: Reuters: Health)
Source: Reuters: Health - December 24, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche enters $1.15 billion licensing deal for Sarepta gene therapy
Roche entered into a $1.15 billion licensing agreement with Sarepta Therapeutics to obtain the right to launch and commercialize Sarepta's investigational gene therapy for Duchenne muscular dystrophy (DMD) outside the United States. (Source: Reuters: Health)
Source: Reuters: Health - December 23, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy
Basel, 23 December 2019 - Roche (SIX: RO, ROG; OTCQX: RHHBY) and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), today announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin),   Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the terms of the agreement, Sarepta will receive an upfront payment of $750million in cash and $400million in equity. In addition, Sarepta is eligible to receive regulatory and sales m ilestones, and royalties on net sales. Roche and Sarepta will equally s...
Source: Roche Media News - December 23, 2019 Category: Pharmaceuticals Source Type: news

Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy
Basel, 23 December 2019 - Roche (SIX: RO, ROG; OTCQX: RHHBY) and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), today announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin),   Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the terms of the agreement, Sarepta will receive an upfront payment of $750million in cash and $400million in equity. In addition, Sarepta is eligible to receive regulatory and sales m ilestones, and royalties on net sales. Roche and Sarepta will equally s...
Source: Roche Investor Update - December 23, 2019 Category: Pharmaceuticals Source Type: news

Rare diseases: customizing cures, patient by patient
Yale geneticist Monkol Lek, Ph.D., who has muscular dystrophy, wants to cure a rare form of the disease. (Source: Yale Science and Health News)
Source: Yale Science and Health News - December 20, 2019 Category: Universities & Medical Training Source Type: news

In surprise decision, US approves muscular dystrophy drug
The U.S. Food and Drug Administration has approved a second drug to treat muscular dystrophy, the debilitating disease that causes loss of movement and early death (Source: ABC News: Health)
Source: ABC News: Health - December 14, 2019 Category: Consumer Health News Tags: Health Source Type: news