Adolescent patients with obstructive hypertrophic cardiomyopathy (HCM) who received the drug mavacamten saw a significant ...

EMA’s panel backs a targeted therapy, which reduces lymphadenopathy and restores B-cell populations in patients with activated phosphoinositide 3-kinase delta syndrome.

First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved ...

Crispr Therapeutics AG looks set for 2026 revenue inflection as Casgevy ramps; strong efficacy, undervalued shares, robust ...

The FDA has given accelerated approval to Avlayah as a treatment for neurologic manifestations of Hunter syndrome, also known ...

Unique in its focus on adolescents, the trial shows the cardiac myosin inhibitor safely reduces LVOT in the pediatric ...

Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of Follow-u ...

First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved medicine in emerging new class of biotherapeutics that leverag ...

Absci Corporation downgraded to Hold as ABS-101 stalls and focus shifts to ABS-201; cash runway to H1 2028, but risks persist ...

India, March 27 -- Delhi Chief Minister Rekha Gupta on Thursday distributed bicycles to 1,100 schoolgirls in Shalimar Bagh.