Duchenne Muscular Dystrophy Trial has Gotten the Go Ahead from the FDA to Continue Dosing

Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…

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Update On Newborn Screening Efforts for Duchenne Muscular Dystrophy
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Update On Newborn Screening Efforts for Duchenne Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…

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New Phase 1 Study for Becker Muscular Dystrophy Treatment
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New Phase 1 Study for Becker Muscular Dystrophy Treatment

Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…

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This Drug Could Boost Duchenne Muscular Dystrophy Treatment
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This Drug Could Boost Duchenne Muscular Dystrophy Treatment

According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…

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Ataluren Halts Disease Progression in Patients with nmDMD
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Ataluren Halts Disease Progression in Patients with nmDMD

This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…

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This New Collaboration Aims to Create Gene Therapies for Duchenne Muscular Dystrophy
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This New Collaboration Aims to Create Gene Therapies for Duchenne Muscular Dystrophy

According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…

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Phase 3 SIDEROS Trial for DMD Drug Idebenone Fails
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Phase 3 SIDEROS Trial for DMD Drug Idebenone Fails

While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…

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Duchenne Muscular Dystrophy Gene Therapy Gets FDA Fast Track Designation
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Duchenne Muscular Dystrophy Gene Therapy Gets FDA Fast Track Designation

According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…

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Positive Data Announced for SRP-9003, A Gene Therapy for LGMD2E
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Positive Data Announced for SRP-9003, A Gene Therapy for LGMD2E

  At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…

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Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon
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Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon

As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…

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Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model
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Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model

According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…

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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice
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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice

According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…

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Enrollment Begins in Trial of Duchenne Muscular Dystrophy Treatment

According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…

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Researchers Produce End-to-End X Chromosome DNA sequence

  As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…

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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy
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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy

A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…

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MRFF Funding Promotes Treatment Development for Patients with Rare Diseases
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MRFF Funding Promotes Treatment Development for Patients with Rare Diseases

  When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…

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Study: Gene Therapy as a Treatment for Duchenne Muscular Dystrophy
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Study: Gene Therapy as a Treatment for Duchenne Muscular Dystrophy

According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…

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A New Partnership will Advance Exosome-Based Gene Therapy for Neuromuscular Diseases
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A New Partnership will Advance Exosome-Based Gene Therapy for Neuromuscular Diseases

By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…

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ICYMI: SRP-9003 Study Shows Positive Results for Treating LGMD Type 2E
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ICYMI: SRP-9003 Study Shows Positive Results for Treating LGMD Type 2E

  In a recent press release, biotechnology company Sarepta Therapeutics announced positive findings from their clinical trial studying SRP-9003 as a treatment for limb-girdle muscular dystrophy (LGMD) type 2E. This…

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New Data Gives Hope for a Limb-Girdle Muscular Dystrophy Gene Therapy
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New Data Gives Hope for a Limb-Girdle Muscular Dystrophy Gene Therapy

According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…

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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary
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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary

  According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…

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Those with Disabilities and Rare Medical Conditions in Canada are Excluded from Financial Relief During Covid-19

Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…

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New Pediatric Certified Duchenne Muscular Dystrophy Care Center in Texas
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New Pediatric Certified Duchenne Muscular Dystrophy Care Center in Texas

Parent Project Muscular Dystrophy is a nonprofit that aims to find a cure for Duchenne muscular dystrophy. One of their programs, the Certified Duchenne Care Center Program (CDCCP), has recently…

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