Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD
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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD

In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…

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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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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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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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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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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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