Trial
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Gene therapy injection into the eye shows promise for treating inherited blindness caused by RPGR gene faults in men.
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.
Gene therapy injection into the eye shows promise for treating inherited blindness caused by RPGR gene faults in men.
Global registry tracking Pompe disease progression and treatment outcomes in patients to improve diagnosis, monitoring, and therapeutic guidance.
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to…
Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in…
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,…