Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...
7hon MSN
This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
Key market opportunities include the surge in biopharmaceutical outsourcing for nucleic acid production, driven by demand for ...
Lilly inks gene therapy deal with Seamless Therapeutics to develop programmable recombinase treatments targeting genetic hearing loss worldwide.
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...
What if a cup of coffee could help treat cancer? Researchers at the Texas A&M Health Institute of Biosciences and Technology ...
The FDA granted orphan drug designation to OpCT-001 for the treatment of retinitis pigmentosa, according to a press release ...
Regenxbio (RGNX) shares rallied 5.4% in the last trading session to close at $11.6. This move can be attributable to notable volume with a higher number of shares being traded than in a typical ...
Eli Lilly plans to expand its hearing loss therapy pipeline by collaborating with Seamless Therapeutics to apply the company’s next-generation gene-editing approach, based on programmable recombinases ...
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