EU green light to IntraBio’s AQNEURSA for rare neurological disease Niemann-Pick Type C

The approval is grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients

Novo Medi Sciences unveils India’s next-generation varicella vaccine NEXIPOX PLUS

This innovation enhances viral stability, preserves potency, and reduces allergic reactions and antigen crystallisation-related issues

Teva’s AJOVY gains spotlight as first preventive migraine treatment for kids

The findings, from the SPACE study, paved the way for FDA approval of AJOVY for pediatric patients aged 6-17 years weighing 45 kilograms

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