CAP-1002, a cell therapy being developed by Capricor Therapeutics to treat Duchenne muscular dystrophy (DMD), has won a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). RMAT status is given to regenerative medicine therapies that show potential to treat serious conditions lacking available therapies.
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European and Japanese researchers used an artificial chromosome to generate the dystrophin protein that’s missing in Duchenne muscular dystrophy. The chromosome delivered a two-gene package capable of producing the missing protein to a mouse model of the disease. The team also used it in a lab setting involving human cells.
The recently formed company Skyhawk Therapeutics announced it has obtained $8 million in seed funding to develop RNA-targeting small molecules for the treatment of over 50 diseases, including muscular dystrophy. The investment is led by “major family and biotech investors,” including Tim Disney, Alexandria Venture Investments, and the Duke of Bedford, among others,…
This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…
An advisory committee of the European Medicines Agency (EMA) has issued another negative recommendation after re-examining a request to extend Raxone (idebenone) for use in treating Duchenne muscular dystrophy (DMD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued its first negative opinion of the request in…
Ezutromid significantly reduced muscle damage in patients with Duchenne muscular dystrophy (DMD) after 24 weeks of treatment in a Phase 2 trial, Summit Therapeutics recently announced. DMD is a severe type of muscular dystrophy caused by the absence of dystrophin, a protein…
A dysfunctional cerebellum leads to implicit learning deficits in children with Duchenne muscular dystrophy (DMD), Italian researchers have suggested. Their study, “Implicit learning deficit in children with Duchenne muscular dystrophy: Evidence for a cerebellar cognitive impairment?” appeared in the journal PLoS ONE. DMD is an…
Researchers have found that cognitive function, including the ability to multitask and solve problems, may be impaired in boys with Duchenne muscular dystrophy (DMD) — even in those with normal and above average IQs. Their study, “Cognitive profile in Duchenne muscular dystrophy boys without intellectual disability: The role of executive functions,”…
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Young Boy Becomes First DMD Patient to Receive Investigational Systemic Microdystrophin Gene Therapy
The first Duchenne muscular dystrophy (DMD) patient has received systemic microdystrophin gene therapy as part of a Phase 1/2 clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The trial (NCT03375164), currently recruiting participants, will test the safety and efficacy of a single dose of intravenous microdystrophin gene therapy…
A global Phase 3 trial of givinostat — an investigational treatment aiming to boost the muscle regeneration capacity in Duchenne muscular dystrophy patients — is recruiting participants across North America and Europe. The trial is looking to recruit boys, older than six years, who are still ambulatory and on a stable…
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