A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
A Massachusetts man became the first person to try intranasal foralumab under an expanded access program for people with ...
While Illinois joins states challenging cuts to medical research funding, an Illinois State University lab supported by the ...
The Next Chapter is keeping the tears coming this season. Leah Messer is having a tough time getting through to daughter Ali ...
Five oculopharyngeal muscular dystrophy (OPMD) patients have now been treated with the gene therapy candidate BB-301 in a ...
Enrollment of healthy volunteers for a Phase 1 trial testing Duchenne muscular dystrophy (DMD) treatment candidate SAT-3247 ...
At four-years-old, William Moore’s muscles are already beginning to deteriorate. Something as small as a slight gust of wind and sometimes even just thin air is enough to knock William over. Diagnosed ...
By Balambal Suryanarayanan CRISPR-Cas9 is a repurposed gene editing tool that allows scientists to cut, replace, and insert pieces of DNA in precise regions along the strand. Courtesy of NIH Image ...
The global Gene Editing Market was valued at $5.1 billion in 2022 and is projected to reach $16.2 billion by 2030, growing at ...
Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...
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