Topic
Rare Disease
3 stories about Rare Disease that passed the news check, newest first.
Research ·
Science solves genetic mystery behind fatal brain disorder in babies and children
Researchers at KAUST and King Faisal Specialist Hospital and Research Center identified a previously unknown genetic cause of a fatal newborn brain disorder.
Medical Xpress - latest medical and health news stories
Research ·
Pharmacological chaperone treatment with Cystadane in aspartylglucosaminuria: an open-label, phase Ib/II trial
An open-label phase Ib/II clinical trial tested Cystadane (betaine) as a pharmacological chaperone in aspartylglucosaminuria.
nature.com
News ·
Another Option for Rare Benign Soft-Tissue Tumor Wins FDA Approval
The FDA approved the small-molecule inhibitor pimicotinib, sold as Pimjetmi, for selected patients with symptomatic tenosynovial giant cell tumour (TGCT).
MedPageToday.com - medical news for physicians