Bristol Myers Squibb reports positive Phase 3 results for Camzyos in adolescents with oHCM

The study showed a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo

GRIN Therapeutics launches global Phase 3 trial for groundbreaking GRIN-NDD therapy

GRIN-NDD is one of the most complex neurodevelopmental disorders with limited treatment options today

Rapid-acting ADHD treatment Azstarys now approved in China

ADHD is a common, chronic neurodevelopmental disorder that often begins in childhood and can persist into adulthood

FDA nod to first-ever treatment for deadly post-transplant complication TA-TMA

The decision positions Yartemlea as the first and only approved inhibitor of the lectin pathway of complement

US House passes Bill reauthorizing FDA rare disease incentive program

Neurocrine’s Valbenazine fails to show benefit in largest dyskinetic cerebral palsy trial

Adverse events were consistent with valbenazine’s established safety profile

Abbott wins FDA & CE nod for groundbreaking premature infant heart device

The system is intended for some of the tiniest patients—premature infants weighing as little as two pounds

FDA accepts Sentynl Therapeutics CUTX-101 NDA resubmission

If approved, CUTX-101 will be the first and only FDA approved treatment available for Menkes disease

Sanofi secures two breakthrough nods in China for rare blood disorders

Qfitlia is the first antithrombin (AT)-lowering therapy approved for routine prophylaxis in hemophilia

Caplin Steriles gets FDA final approval for Acetaminophen Injection, infusion bags

Acetaminophen Injection is indicated for the management of mild to moderate pain in adult and pediatric patients 2 years and older