Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...

Enrollment Complete in Cohorts 1 and 2 of MyPEAK™-1 Phase 1b/2 Trial of TN-201 for MYBPC3-associated HCM; Positive DSMB ...

Cash runway extended into 2028; expected to enable multiple meaningful clinical data read-outs -- APOE program added to ...

At only 6 months, Gerard and Kaitlin Norton’s daughter Madeline was diagnosed with PKAN, a rare neurodegenerative disorder ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

From alveolar injury to extracellular matrix accumulation, the complex biology of pulmonary fibrosis is being unraveled ...

In this week’s edition of InnovationRx, we look at a biotech entrepreneur treating a rare genetic disease, new tech aimed at ...

Novo Nordisk will advance its acquired ATTR amyloidosis with cardiomyopathy monoclonal antibody (mAb) to Phase III trials.