Couple donates $11 million to Lurie Children’s Hospital
A couple with deep ties to the Chicago area is donating $11 million to Lurie Children’s Hospital in…
Pharma sounds alarm over EU plans to procure novel medicines – POLITICO
But the pharmaceutical industry argues that any changes should be made cautiously and with clear guidelines for everyone…
GeneDx to Partner with Florida’s Sunshine Genetics Network, Launching Nation’s First State-Backed Genomic Newborn Screening Program
Florida Institute for Pediatric Rare Diseases and Representative Adam Anderson ushering in a new era of preventative care…
NLRP3 inflammasome and hearing loss: from mechanisms to therapies | Journal of Neuroinflammation
Sensorineural hearing loss (SNHL) affects approximately 1.5 billion people globally. Approximately 430 million of these patients require rehabilitation…
Knowing patient’s genetic make-up may change the way medicines are prescribed
SINGAPORE – Not all medicines work the same way on every patient, and part of the answer lies…
Acadia Pharmaceuticals drug for rare genetic condition fails late-stage study
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
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…