Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key ...

Clopidogrel is a prescription drug used to reduce the risk of heart attack and stroke. Learn about the common, mild, and ...

Altuvoct (efanesoctocog alfa) was approved in the U.K. as a treatment for patients with moderate or severe hemophilia A ages ...

Alnylam awaits FDA decisions on Amvuttra & Fitusiran next month, poised to boost revenue, profitability, and share growth.

Antibody therapy Mim8 was found to be safe and effective for controlling bleeds in children with hemophilia A in a Phase 3 ...

Pfizer terminated partnership with Sangamo on hemophilia A gene therapy despite positive Phase 3 data. Find out why SGMO ...

Using “cure” to refer to “obtaining a normal life” also fits — if you ignore previously lost education, career opportunities ...

Novo Nordisk today announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with haemophilia A with and without inhibitors. The trial initially assessed ...

Random bruising after workouts occurs due to broken blood vessels under the skin, intense exercise, nutrient deficiencies, or ...

Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds ...

Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated ...

Gout can occur in the hip joint rarely. In gout, uric acid crystals build up in the joints. There can be sudden, severe pain ...