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Numerous studies show that deflazacort substantially prolongs walking ability in boys with Duchenne muscular dystrophy (DMD), allowing them to lead a longer productive life. The drug also causes less weight gain than other similar medications, a factor scientists believe might be key to maintaining ambulation. Despite its worldwide popularity as…

The second annual Napa in Newport drew more than 420 guests in a sold-out event that raised more than $700,000 to help to find a cure for Duchenne muscular dystrophy. The money will go to CureDuchenne, a nonprofit dedicated to finding a cure for Duchenne. The event was chaired by Darioush and Shahpar…

Scientists have discovered why fetal muscle stem cells are better at regenerating muscle than adult muscle stem cells. These findings open new avenues for the treatment of muscular dystrophies. Researchers from Sanford Burnham Prebys Medical Discovery Institute, based in La Jolla, California, and Orlando, Florida, investigated the properties of different cells in a study…

Tens of thousands of businesses across the U.S. are participating in this year’s 34th annual Muscular Dystrophy Association (MDA) Shamrocks campaign to raise funds for research and support programs to help people with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and other diseases that severely weaken muscle…

The Precision Medicine Initiative was launched in 2015 to spur biomedical research and the development of a more personalized approach to therapy, one based on an individual’s genetic profile and response to treatment. While the initiative largely focuses on genetically based cancers, a University of Wisconsin-Madison review points out that Emery–Dreifuss muscular…

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…