News

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is a double-blind, placebo-controlled, multi-center Phase 3 clinical trial (NCT02500381) with an open-label extension period, evaluating the two drugs’…

A gene therapy treatment may restore the genetic mutations of myotubular myopathy, a type of muscular dystrophy that affects the skeletal muscles. In a previous study, a team of researchers at the University of Washington School of…

A new clinical-stage biotech, Myonexus Therapeutics, is developing gene therapies for limb-girdle muscular dystrophies (LGMDs). Launch plans include the accelerated development of five investigational gene therapies that have the potential to become the first standard-of-care therapies for LGMDs. Myonexus’ pipeline includes three clinical-stage gene therapy programs (LGMD2E, LGMD2D, and…

The U.S. Food and Drug Administration (FDA) scheduled a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting with PTC Therapeutics to review the company’s new drug application (NDA) for Translarna (ataluren) to treat patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The meeting will take place on Sept. 28, the company…

Genetic screening for the presence of mutations that characterize Duchenne and Becker muscular dystrophies (DMD and BMD) in women before conception can help determine the risk their offspring will develop either disease, according to a Polish study published in the Journal of Obstetrics Gynaecology Research. The study, “Prenatal diagnosis of Duchenne…

Capricor Therapeutics will hold a phone meeting with the U.S. Food and Drug Administration (FDA) this month to discuss potential product registration strategies for CAP-1002, a cardiac cell therapy being developed to treat Duchenne muscular dystrophy (DMD). CAP-1002 is produced from a healthy donor’s heart tissue, grown in a lab and stored…

MoveDMD (NCT02439216) is a three-part, ongoing clinical trial investigating an oral drug — edasalonexent (CAT-1004) — as a potential therapy in boys, ages 4 to 7, with Duchenne muscular dystrophy (DMD). The drug targets NF-kB, a protein activated in DMD and shown to promote the disease by driving inflammation, muscle degeneration,…

An investigational therapy to treat congenital myotonic dystrophy, AMO-02 (tideglusib), has been granted fast track designation by the U.S. Food and Drug Administration (FDA), AMO Pharma has announced. Myotonic dystrophy (DM) is the most common form of muscular dystrophy (MD). DM type 1 is the consequence of a…

A proposed U.S. database of children with complex medical problems, including muscular dystrophy (MD), has raised concern at a respected public policy institute. Researchers at the Houston institute contend that the database could be used to discriminate against the children and their families, in particular to deny them health insurance. Dr.

The U.S. Food and Drug Administration (FDA), which in September 2016 approved Serepta Therapeutics’ Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen), now finds itself the subject of a lawsuit aimed at forcing the FDA to turn over information about the approval process. Charles Seife, a journalism professor at New…