Trial
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
What is being tested: This is a natural history study tracking disease progression in Friedreich ataxia patients over time across multiple international…
No directly tagged education is live for Rare Diseases yet. Use the specialty context below plus relevant cross-specialty education while the dedicated hub is expanded.
Use the specialty context and cross-specialty learning below while dedicated modules, podcasts and webinars are added.
What is being tested: This is a natural history study tracking disease progression in Friedreich ataxia patients over time across multiple international…
Gene therapy trial testing safety and effectiveness of DB-OTO for hearing loss caused by otoferlin gene mutations in children and adults.
What is being tested: Pegcetacoplan (a complement C3 inhibitor) effectiveness and safety in real-world clinical practice for patients with C3 glomerulopathy (C3G)…
England is introducing newborn screening for spinal muscular atrophy (SMA), a genetic neuromuscular disorder, as part of a major national study programme…
recorded webinar focused on SGLT2 Inhibitors in diabetes and metabolic medicine. Use the recording with the slide outline to review mechanism, evidence,…
Recorded webinar focused on JAK inhibitors in rheumatology and immune-mediated inflammatory disease. Reviews JAK-STAT signalling, patient selection, ORAL Surveillance safety signals, cardiovascular…
recorded webinar focused on GLP-1 Agonists in diabetes and metabolic medicine. Use the recording with the slide outline to review mechanism, evidence,…