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…
The Muscular Dystrophy Association, in collaboration with the Lewin Group, has calculated the average cost of caring for three of the most common neuromuscular disorders. MORE:Â Study reveals wide global disparities in prevalence, burden, cost, and treatment of DMD. The study looks at both medical and…
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…
This video from Muscular Dystrophy UK is all about 16-year-old Karis Williamson from Inverness, Scotland. Karis used to love going to school, but as she got older, she found it frustrating and felt like an unvalued member of the class. Feeling left out and like a burden, she…
A neuromuscular disorder diagnosis will have a profound effect on the whole family. Younger siblings often don’t fully understand what a neuromuscular disorder is or what it means for their brother or sister. MORE: How to talk to your child about their neuromuscular disorder. It’s best to be upfront and…
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…
This video from Muscular Dystrophy UK is all about Hayleigh Barclay. Hayleigh is a 27-year-old woman from South Ayrshire, Scotland, who has spinal muscular atrophy (SMA) type 2. MORE: Teen with muscular dystrophy launches YouTube gaming channel to highlight the disease. Hayleigh’s passion is music and film.
Healthcare costs for patients with Duchenne muscular dystrophy (DMD) are about 10 times those of healthy people, and increase as patients age, according to a new analysis of claims data. The figures, which may aid in healthcare planning and evaluating costs of emerging treatments, were — for the first time —…
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