The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare ...
Two experts discussed innovative trial designs in rare disease research, common challenges and the potential of AI to enhance research.
People with Sweet's syndrome often wake up with a fever, body aches, and blisters on their neck, limbs and face. Also known ...
Baylor College of Medicine has received new funding from the National Institutes of Health for two consortia in the Rare ...
Working with agency partner DCX Growth Accelerator, GeneDX takes direct aim at the issue of underdiagnosis of pediatric rare diseases.
Rocket Pharmaceuticals' strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was ...
Patients and medical experts met on Emory’s campus to discuss a little-known disease that impacts thousands of Americans.
SIOUX FALLS, S.D. (Dakota News Now) - Erin Darling knows firsthand what it’s like to live with a rare disease.
Postoperative haemorrhage can appear unexpectedly even after routine appendicectomy, especially in patients with ...
Cade was diagnosed with a rare, neurodegenerative disease when he was 2½. Now 4, Cade has been receiving treatment for the past few years — crucial to keep him strong — but the program he's in has ...
A woman with a rare disease has thanked Sark's doctor and community responders for an "amazing evacuation process" which ...
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