FDA’s Rare Disease Innovation Hub Releases Strategic Agenda: Implications for Stakeholders in 2025
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...
Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.
TD Cowen began coverage on Palvella Therapeutics shares, assigning a Buy rating and setting a price target of $44.00. The stock, currently trading at $13.89, has already shown strong momentum with a ...
SOUTH SAN FRANCISCO, Calif. - Quince Therapeutics, Inc. (NASDAQ: QNCX), a biotech firm focusing on rare diseases with a current market capitalization of $67.76 million, has received a Notice of ...
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
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