Genetics

Findings mark a significant breakthrough in understanding how MS progresses

Challenges of drug development in fibrodysplasia ossificans progressiva on full display

Large study supports wider screening of family members of probands

Metreleptin doses several times higher than recommended brought weight down to near normal

Success observed in usually contraindicated Marfan, Loeys-Dietz syndromes

Future treatments don't have to be limited to slowing disease, case series suggests

When disparities are considered, researchers suggest it can be

FDA's decision marks first approval for a redosable gene therapy

CBER director says "moment is tender for gene therapy," sees opportunity to "salvage" treatments

Finding challenges previous assumptions about disease

Findings may bolster interest in BTK inhibitors for autoimmune diseases

Patients ages 2 to 5 years now eligible for the triple therapy

Health news and commentary from around the Web gathered by MedPage Today staff

Fewer actionable mutations, less likely to meet genomic criteria for immunotherapy