News

Ways of better addressing the high cost of breakthrough treatments that either are or might soon be available for a range of chronic and rare diseases, from certain cancers to Duchenne muscular dystrophy (DMD) or Becker’s muscular dystrophy (BMD), was the subject of recent research — with the authors suggesting that healthcare…

Capricor Therapeutics, Inc., a clinical-stage biotechnology company, has treated the first Duchenne muscular dystrophy patient with related cardiomyopathy with its drug candidate CAP-1002, and is continuing to enroll people in its HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) Phase I/II clinical trial (ClinicalTrials.gov Identifier: NCT02485938). CAP-1002 is a proprietary allogeneic,…

PTC Therapeutics, Inc. announced Wednesday that it has received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) with regard to its New Drug Application (NDA) for ataluren (trade name, Translarna), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy…

One hundred and nine members of the U.S. House of Representatives signed a letter to the U.S. Food and Drug Administration (FDA) calling for the agency to immediately increase efforts to mobilize all available tools, resources, and authorities to advance new and more effective treatments for Duchenne muscular dystrophy. The bipartisan appeal…

Tarix Orphan LLC recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127, the company’s lead candidate for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD). Early-onset LAMA2-related muscular dystrophy (MD) occurs in nearly one in every 30,000 people and is responsible for…

Pfizer, in collaboration with Newcastle University, reports finding four serum biomarkers for three types of muscular dystrophy. The markers may be valuable for monitoring disease progression and treatment response in both preclinical and clinical studies. Researchers analyzed previously collected serum samples from three types of muscular dystrophies — 38 Becker muscular dystrophy (BMD) and…

UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…

A discovery clears up the confusion about the molecular factors involved in muscle repair. The research, published in the journal e-life, shows that a protein complex containing the factors TBP and TFIID is essential to activate genes responsible for adult muscle tissue regeneration. Adult muscles contain stem cells that can be activated…

Catabasis Pharmaceuticals announced it will present data on CAT-1004 at the XIV International Conference on Duchenne and Becker Muscular Dystrophy to be held in Rome, Italy, on Feb. 13-14. Joanne Donovan, chief medical officer of Catabasis, will talk about the drug, now in clinical testing, and the design of the MoveDMD…

Sarepta Therapeutics, a developer of RNA-targeted treatments, announced that the U.S. Food and Drug Administration (FDA) is extending by another three months its review of the company’s New Drug Application (NDA) for eteplirsen, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A reason for the extension was…