Solid Biosciences Inc. (NASDAQ:SLDB) develops therapies for neuromuscular and cardiac diseases in the US. In addition, the ...
A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...
A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.
Average Upside Potential as of December 10: 104.08% ...
Investor attention has returned to Sarepta Therapeutics amid reports of the biotech firm's vigorous legal strategy to protect ...
Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...
For nearly a decade, Solid has been a staunch supporter and a steering committee member of Parent Project Muscular Dystrophy’s (PPMD) committee dedicated to implementing newborn screening for Duchenne ...
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.
Last 28 March 2025, the European Commission decided not to renew the conditional marketing authorisation for Translarna (ataluren) in Duchenne’s disease due to lack of efficacy after 11 years in the ...
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