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

Investors pile in as Cordance secures oversubscribed seed round for brain-cancer tech

The fresh capital will propel Cordance into its first-in-human clinical trial

Novartis scores FDA nod for game-changing SMA treatment

Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA

FDA investigates fatal case linked to rare blood disorder drug Adzynma

Adzynma, approved for both adults and children with cTTP, is a recombinant enzyme therapy designed to replace the deficient ADAMTS13 enzyme

Mesoblast teams with NIH to launch pivotal trial of life-saving cell therapy in adults with severe aGvHD

The therapy -- Ryoncil (remestemcel-L-rknd) -- will be tested in adults with severe steroid-refractory acute graft-versus-host disease

FDA nod to Koselugo for adults with Rare NF1 tumors, expanding life-changing treatment

Alembic announces USFDA final approval for Dasatinib Tablets

The approved ANDA is therapeutically equivalent to the reference listed drug (RLD), Sprycel Tablets