FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.

Blueprint Medicines, a Sanofi company, today announced AYVAKIT(R) (avapritinib) data showing clinically meaningful outcomes and a w ...

Researchers develop a single genome-editing strategy to treat multiple disorders caused by nonsense mutations, promising efficient and cost-effective therapies.

Learn more about whether IDEAYA Biosciences, Inc. or Travere Therapeutics, Inc. is a better investment based on AAII's A+ ...

Company entering 2026 with the potential to deliver multiple value-driving milestones, including data readouts from ARV-102, ARV-806, and ARV-393, with cash runway into second half of 2028 - ...

NGGT002 (NGGT), JNT-517 (Jnana Therapeutics), Pegvaliase (BioMarin), and others under investigation, the phenylketonuria treatment landscape is expected to undergo substantial transformation between ...

Oppenheimer 36th Annual Healthcare Life Sciences Conference February 26, 2026 1:20 PM ESTCompany ParticipantsTom Frohlich ...

Discusses Positive Phase 3 PROPEL 3 Results for Infigratinib in Children With Achondroplasia February 12, 2026 8:00 ...

Immunocore reports fourth quarter and full year 2025 financial results and provides a business update KIMMTRAK (tebentafusp-tebn) Q4 net sales of $104.5 million and $400.0 million for full year 2025; ...

Once a defining tree of eastern forests, the American chestnut was nearly erased by a foreign fungus within a few human generations. A genetically engineered line known as Darling 54 has now pushed ...

DC Comics has plenty of strange characters to choose from, but these are the weirdest.

Two local bivalves — meaning “two shells” and referring to mollusks like oysters and clams — have developed contagious cancers: the eastern soft-shell clam, which is non-native and arrived from the ...