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

Capricor's cell therapy shows breakthrough results in Duchenne muscular dystrophy trial

It's a development that marks a potential breakthrough for people living with the rare and debilitating disease Duchenne muscular dystrophy (DMD)

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies

Belite Bio reports landmark phase 3 success for Stargardt Disease therapy

Seeing well-controlled Phase 3 data that shows a marked slowing of lesion growth in Stargardt disease is deeply encouraging

Renalys Pharma reports major phase III success for Sparsentan in Japanese IgA nephropathy patients

The study, which enrolled 35 Japanese patients, evaluated the percent change from baseline in the 24-hour urine protein-to-creatinine ratio