Rare Diseases
Guidelines, trial intelligence, clinical news, drug science and education for Rare Diseases.
Trial Radar
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…
TrialPompe Disease Registry Protocol
Global registry tracking Pompe disease progression and treatment outcomes in patients to improve diagnosis, monitoring, and therapeutic guidance.
PHASE3A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males…
PHASE3Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD)
Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity…
Relevant Mechanisms
Drug science mapping in progress
Mechanism explainers will appear here as they are mapped to Rare Diseases.
CPD, Podcasts & Webinars
No specialty-tagged education live yet
Rare Diseases podcasts, webinars and CPD modules will appear here as new education is published and tagged.
Useful starting points in Rare Diseases
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…
TrialPompe Disease Registry Protocol
Global registry tracking Pompe disease progression and treatment outcomes in patients to improve diagnosis, monitoring, and therapeutic guidance.
TrialA Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males…
Education that still supports this area
SGLT2 Inhibitors: From Glycaemia to Heart Failure and Chronic Kidney Disease
recorded webinar focused on SGLT2 Inhibitors in diabetes and metabolic medicine. Use the recording with the slide outline…
WebinarJAK Inhibitors: Updated Safety Signals, Patient Selection and Post-ORAL Surveillance
Recorded webinar focused on JAK inhibitors in rheumatology and immune-mediated inflammatory disease. Reviews JAK-STAT signalling, patient selection, ORAL…
WebinarGLP-1 Agonists: Semaglutide, Tirzepatide and the Cardiovascular Revolution
recorded webinar focused on GLP-1 Agonists in diabetes and metabolic medicine. Use the recording with the slide outline…
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