Will Forte joins Teva Pharmaceuticals to shine spotlight on Huntington’s disease

HD causes cognitive, behavioral, and physical challenges, including involuntary movements known as HD chorea, which can disrupt daily life

Bausch Health’s RED-C phase 3 trials fall short, company committed to liver disease research

The trials, which enrolled over 1,000 patients across 398 sites in 17 countries, showed that the drug was safe and well-tolerated—but ultimately failed to deliver the hoped-for clinical results

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

FDA nod to Boehringer Ingelheim's heart disease treatment for dogs 'Vetmedin'

VETMEDIN (pimobendan) chewable tablets and VETMEDIN solution (pimobendan oral solution) can delay the onset of congestive heart failure (CHF) in dogs

FDA nod to first-ever treatment for fatal pediatric Menkes disease

Subcutaneous Saphnelo cuts lupus disease activity in phase III trial

SLE remains a life-threatening autoimmune disease, with patients facing an elevated risk of early mortality and long-term organ damage

Savara resubmits FDA application for potential treatment in rare lung disease patients

The resubmission of BLA for MOLBREEVI names Fujifilm as the drug substance manufacturer

INOVIO’s DNA therapy for rare throat disease gets FDA review

INOVIO remains confident in the therapy’s benefits and intends to meet with the FDA to discuss maintaining eligibility under this pathway

US House passes Bill reauthorizing FDA rare disease incentive program

AB Science secures US patent for Masitinib in sickle cell disease till 2040

Masitinib targets mast cells, which play a critical role in severe forms of SCD and its complications, including vaso-occlusive crises, acute chest syndrome, and pain