Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Chronic malabsorption syndromes such as Crohn disease, chronic pancreatitis, and short bowel syndrome are associated with an increased risk for AMD.

Investigational RGX-202 continues to demonstrate evidence of positively changing disease trajectory for Duchenne Pivotal dose participants exceeded external controls across functional ...

Testosterone replacement therapy before total knee arthroplasty was linked to higher risks for systemic and periprosthetic complications.

Updated mRNA-1273 vaccine was effective against severe COVID-19 outcomes in older and high-risk adults during the 2024-2025 season.

Among patients with rheumatoid arthritis-associated interstitial lung disease, there was no significant difference in the ...