This page keeps track of new/upcoming treatment options for muscular dystrophy. What’s in development? What’s promising? What’s available soon? Find out below… Current topics include vamorolone, SRP-9001, ANXA6, edasalonexent, CAT-1004, gene therapy, stem cells, anti-CD45RC, and more.
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News
- Dystrogen Therapeutics Announces That Treatment With Dystrophin Expressing Chimeric (DEC) Cells Improves Cardiac Function in Preclinical Duchenne’s Study Nov 5, 2019
- In Lab, SLU Research Halts Toxic Protein Linked to Muscular Dystrophy Oct 23, 2019
- Vamorolone Designated Promising Innovative Medicine (PIM) for Treatment of Duchenne Muscular Dystrophy by the UK MHRA Oct 21, 2019
- Gene therapy for Duchenne muscular dystrophy safely preserves muscle function Oct 7, 2019
- Catabasis Pharmaceuticals Announces the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne Muscular Dystrophy has Exceeded Target Enrollment Sep 30, 2019
- Stem cells derived from patients could lead to new treatments for congenital muscular dystrophies Sep 30, 2019
- Catabasis Pharmaceuticals to Present the MoveDMD Trial of Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy at the 24th International Congress of the World Muscle Society Sep 27, 2019
- Researchers developing new ‘DNA stitch’ to treat muscular dystrophy Sep 25, 2019
- CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy Sep 18, 2019
- CRISPR fix in mice may lead to muscular dystrophy therapy Sep 18, 2019
- Adults Living with DMD, Parents and Patients, Voice ‘Cautious Optimism’ for Gene Therapy in Interviews Aug 28, 2019
- Help may be on the way for people with muscular dystrophy Mar 25, 2019
- Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial Mar 6, 2019
- New drug for Duchenne muscular dystrophy clears phase 1 clinical trial testing in boys Feb 21, 2019
- New insight into use of cell replacement therapies to treat muscular dystrophies Feb 13, 2019
- Gene therapy cassettes improved for muscular dystrophy Feb 1, 2019
- Discovery points to innovative new way to treat Duchenne muscular dystrophy Jan 31, 2019
- Doctors hail breakthrough in muscular dystrophy treatment Aug 30, 2018
- Gene editing of dogs offers hope for treating human muscular dystrophy Aug 30, 2018
- Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy Jul 9, 2018
Research
- Stem cell-based therapies for Duchenne muscular dystrophy Oct 23, 2019
- Targeting NRIP (gene name DCAF6) could be a powerful new approach to treat myocardial dysfunction in limb-girdle muscular dystrophy Oct 17, 2019
- Coenzyme Q10 supplementation acts as antioxidant on dystrophic muscle cells Oct 16, 2019
- Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models Oct 7, 2019
- Surrogate gene therapy for muscular dystrophy Oct 7, 2019
- Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies Oct 2, 2019
- Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes Oct 1, 2019
- Recombinant annexin A6 promotes membrane repair and protects against muscle injury Sep 23, 2019
- miRNA Profiling for Early Detection and Treatment of Duchenne Muscular Dystrophy Sep 19, 2019
- CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors: significant potential for developing future therapies for the treatment of DMD Sep 14, 2019
- Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies Sep 11, 2019
- Immunophenotype of a Rat Model of Duchenne’s Disease and Demonstration of Improved Muscle Strength After Anti-CD45RC Antibody Treatment Sep 9, 2019
- The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice Sep 3, 2019
- Diet Modulation Restores Autophagic Flux in Damaged Skeletal Muscle Cells Aug 30, 2019
- Gene Correction of LGMD2A Patient-Specific iPSCs for the Development of Targeted Autologous Cell Therapy Aug 28, 2019
- Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function Aug 26, 2019
- Cardiac Pathophysiology and the Future of Cardiac Therapies in Duchenne Muscular Dystrophy Aug 22, 2019
- Skeletal muscle cell transplantation: models and methods Aug 7, 2019
- Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies Jul 30, 2019
- Dystrophin R16/17 protein therapy restores sarcolemmal nNOS in trans and improves muscle perfusion and function Jul 2, 2019
- (Epi)genetic Modifications in Myogenic Stem Cells: From Novel Insights to Therapeutic Perspectives May 9, 2019
- Duchenne muscular dystrophy awaits gene therapy Apr 4, 2019
- Do porcine Sertoli cells represent an opportunity for Duchenne muscular dystrophy? Mar 26, 2019
- The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy Mar 5, 2019
- Long-range genomic regulators of THBS1 and LTBP4 modify disease severity in duchenne muscular dystrophy Jul 16, 2018
Clinical Trials
- Exploratory Study of NS-089/NCNP-02 in Duchenne Muscular Dystrophy Oct 16, 2019
- A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 (Suvodirsen) in Ambulatory Patients With Duchenne Muscular Dystrophy Oct 7, 2019
- Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) [Completed] Sep 16, 2019
- A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD) Aug 27, 2019
- Gene Transfer Clinical Trial for LGMD2E (Beta-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB Aug 26, 2019
- Microdystrophin Gene Transfer Study in Adolescents and Children With DMD Aug 15, 2019
- A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy Jul 26, 2019
- Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients Jul 26, 2019
- Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA [Completed] Jul 24, 2019
- Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy Jun 5, 2019
- Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy May 8, 2019
- An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Apr 17, 2019
- Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy [Completed] Apr 16, 2019
- A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy Apr 15, 2019
- Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Apr 12, 2019
- An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy [Completed] Apr 12, 2019
- Study of DS-5141b in Patients With Duchenne Muscular Dystrophy Apr 2, 2019