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

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

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

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

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 ...

Excellos brings its donor-to-dose capabilities—including donor management, apheresis, cell characterization, and GMP ...

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

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

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

During the first half of 2025, we achieved target enrollment in our ongoing gene therapy clinical trials of TN-201 for MYBPC3-associated HCM and ...