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Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD). That’s according to new six-month interim data from the Phase 1b/2a FORWARD-53 trial (NCT04906460), which also found….
PTC Seeks FDA Approval for Translarna to Treat Nonsense …
2017年3月7日 · The U.S. Food and Drug Administration (FDA) acknowledged the filing over protest of PTC Therapeutics ’ new drug application (NDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The FDA granted Translarna standard review and assigned a target date of Oct. 24, 2017 for the agency to ...
Genetic Testing and Genetic Counseling - Muscular Dystrophy News
2022年1月6日 · Muscular dystrophy refers to a group of more than 30 conditions characterized by progressive muscle weakness.. Muscular dystrophies are caused by genetic mutations — errors in the “code” contained in a person’s DNA. Genetic testing, as the term suggests, involves analyzing a person’s DNA in order to detect disease-causing mutations.This type of testing typically is done with cells in ...
Life Expectancy - Muscular Dystrophy News
All muscular dystrophies cause muscle weakness, but life expectancy can vary dramatically between types of muscular dystrophy. While some cases may be mild and progress slowly, others can cause severe muscle weakness and disability. Some people with muscular dystrophy may have reduced life expectancy while others have a normal lifespan.
Del-desiran for DM1 wins FDA’s breakthrough therapy designation
2024年5月14日 · The FDA gives the designation to experimental therapies that have the potential to fill unmet medical needs in treating serious conditions. The designation gives del-desiran’s developer Avidity Biosciences access to perks like more frequent communications with the FDA, with the goal of bringing a needed therapy to market faster. “We are pleased that the FDA has granted breakthrough therapy ...
Ullrich Congenital Muscular Dystrophy (UCMD)
Ullrich congenital muscular dystrophy (UCMD) is a rare hereditary muscle condition that manifests at birth or a few months after birth. It belongs to a group of disorders called collagen type 6-related myopathies and characterized by abnormalities in collagen type 6, a major protein that supports skeletal muscles.. Causes. UCMD is caused by a mutation in one of three genes that provide ...
Potential DMD Treatment, ATL1102, Showing Safety and Muscle...
2019年12月18日 · The experimental therapy ATL1102 is safe and continues to show evidence of improved upper muscle strength and function in all nine non-ambulatory boys with Duchenne muscular dystrophy (DMD) in a nearly complete Phase 2 trial in Australia, updated data show. Final results from the open-label study, testing ATL1102 at low dose, is expected in ...
End Of An Era: MDA Telethon Ends Historic Run, Urgent Fight for...
2015年5月6日 · The Muscular Dystrophy Association (MDA) announced May 1 that after careful consideration and analysis of evolving realities pertaining to both television viewing habits and to philanthropic giving, it is time for the organization to discontinue producing and broadcasting the MDA telethon after 58 years on the air, and move beyond its historic Labor Day telethon — most recently named the ...
Defying the Odds of Living with Duchenne, Decade After Decade
2019年2月8日 · When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at age 6 and immediately began taking corticosteroids as well as heart medicine and supplements. Leonardo Feder, 33, reviews his doctoral thesis.
European committee not in favor of renewing Translarna’s approval
2024年2月1日 · A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company ...