The genetic answer to rare diseases
The standard treatment is a liver transplant, but this entails a high risk of complications in infants. Thanks…
World’s first gene therapy for p47 Chronic Granulomatous Disease developed by UCL and GOSH
image: ©UCL UCL and Great Ormond Street Hospital have developed the world’s first gene therapy for p47 Chronic…
Developments in diagnostic and surgical techniques in children with sagittal suture craniosynostosis: a systematic review spanning the last 30Â years | Orphanet Journal of Rare Diseases
Literature search and study selection An initial search using the search terms related to the operative technique yielded…
Rare disease patients caught up in overseas research funding cuts
Mid-April was a nerve-racking time to arrive on the National Institutes of Health campus outside Washington, D.C. — it…
FDA clears way for Sarepta Therapeutics to use Duchenne drug in some patients
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Sarepta Therapeutics’ Elevidys faces ‘arduous’ path back to market
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Global Pharmaceutical Market Projected to Hit $2.2 Trillion by 2029
BCC Research LLC “Driven by Innovation in Biologics and Expanding Therapeutic Applications, the Global Pharmaceutical Drugs Market Continues…
Sarepta will stop shipping Elevidys, Duchenne gene therapy
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Benefit Lunch for Local Baby with Rare Genetic Disorder | Local News
The Bledsoe County Rescue Squad will host a benefit lunch in Pikeville for a local infant diagnosed with…