Impact of Rare Disease Initiatives at EMA and Health Canada Published 2023-10-12 Download video MP4 360p Download video MP4 720p Recommendations 59:23 What Does Patient Focused Drug Development Look Like for Rare Diseases SD 480p 55:00 Webinar: TRxA BioBRIDGe Awards 1:00:19 The Use and Development of DHTs with Patient Advocacy Groups 2:14:29 Rare Neurodegenerative Disease Efforts Under the ACT for ALS 59:39 Understanding Disease Progression Models: What are They, Why are They Useful, & How Are They Applied 41:06 C-Path Roundtable: C-Path, Help4HD Unite to Discuss Advancements in Huntington's Disease Treatments 15:02 CIVM Workshop Day One: Perspective on Translating New Drug Development Tools into Regulatory Use 55:53 SBS Day 2 - Session IV: Clinical Trial Design Considerations 33:26 Clinical Outcome Assessment — Initial Public Discussion 22:13 Dr. Ken Berry presentation: Principles of a Proper Human Diet 56:42 Improving Data Collection for Rare Epilepsies: Case example from the TSC Natural History Database 1:10:15 SBS Day 2 - Session III-b: Disease Prevention in Lysosomal Diseases 43:58 Jon Fox's Trajectory Was Changed by a Second Opinion - It Takes Balls 23:06 8 Unexpected Ways We Self-Harm & How to Stop 40:24 CIVM 2023 Day 2 : Breakout Sessions — Drafting a Qualification Utilizing Sessions 1, 2, and 3 1:09:28 SBS Day 2 - Panel IV-b Considerations for Clinical Trial Design Optimization in Neonates and T1D 17:26 How to Thrive as A Highly Sensitive Person (HSP) 16:54 SBS Day 2 - Session III-A Disease Prevention in T1D and Neonates (Case Study) 58:30 Improving Employment Outcomes: The Critical Role of Financial Education 17:48 Can What You Eat Reverse Kidney Disease? Similar videos 4:00:46 C-Path Rare and Orphan Disease Conference Day Two AM Sessions 56:14 Making Canada’s $1.5 Billion Rare Disease Drug Strategy Work for Patients 51:31 Fireside Chat on Impact on Rare Diseases at FDA 1:00:40 PMPRB NEW Guidelines and Impact on Rare Diseases 2:58:46 Canadian Rare Disease Ecosystem Symposium: Genetic Therapies for Rare Diseases 24:00 Alessandra Ferlini & Nicolas Garnier: Screen4Care 34:01 CORD & RQMO: Rare Disease Drug Strategy Webinar (English) 48:25 C-Path Rare & Orphan Disease Conference Day Three AM Sessions Sept 13, 2023 3:00:25 C-Path Rare & Orphan Disease Conference AM Sessions Sept 11, 2023 10:13 Clinical Trials: A Lifeline for Patients with Rare Disease 04:41 C-Path Rare and Orphan Conference Michelle Campbell Opening Remarks 1:28:28 EURORDIS webinar: Rare disease registries 59:11 Rare Disease market access in the MENA and Nordic regions 1:03:02 Rare Disease Drug Strategy Webinar 1 October 9 2020 2:09:51 A look at rare diseases across the Americas 58:57 CORD Webinar: Lessons for a SMART Rare Drug System 1:04:02 PMPRB: Friend or Foe of Rare Disease Drug Strategy, CORD Webinar, Nov 6, 2020 1:29:33 FDA and Health Canada Regional ICH Consultation Part I 09:12 Orphan Drug Development | Episode 01- What is Orphan Drug? | Rare Disease | Drug Regulatory Affairs More results