Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment ...

Treatment Market is projected to grow at a healthy rate of around 13% by 2026. This growth is primarily driven by the ...

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and ...

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and ...

KANSAS CITY, Mo. (KCTV) - One resilient metro baby is thriving after a rare genetic disorder diagnosis.

Parents Speak Out After Motorists Target Son with Disabilities at School Bus Stop School Transportation News Wire Reports ...

Family health history is more than just a story—it's a roadmap. As genetics and technology converge, understanding our DNA is ...

As we're approaching the holidays, there is some work being done ahead of time to try and make one little boy's Christmas ...

Grace Nelson, a third-year University of Iowa student from Sioux City, Iowa, was diagnosed with spinal muscular atrophy at 18 ...

CAMBRIDGE, Mass., Nov. 14, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) has completed the acquisition of Alcyone Therapeutics, a clinical-stage biotechnology company known for its innovative ...

The ABC can reveal that in July last year, Queensland Health department became aware of "inconsistencies" in records of the newborn blood spot screening program.