How Exon Skipping Works Published 2024-05-16 Download video MP4 360p Download video MP4 720p Recommendations 43:55 2024 FUTURES - Therapies to Preserve and Protect Muscle 44:41 CureDuchenne Webinar W/ italfarmaco | Community Update with ITF Therapeutics: Introducing DUVYZAT™ 18:05 2024 FUTURES Welcome 12:45 Estiramientos diarios para la distrofia muscular de Duchenne 06:01 Rolling Through the Magic: Exploring Disneyland in a Power Wheelchair 18:32 A World Reimagined: The Transformative Power of Three Simple Words | Ceylan Boyce | TEDxUWE 15:51 WEBINAR: PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024 11:37 2024 FUTURES - CureDuchenne LINK - What We Are Learning Through Your Participation 17:18 Low Sodium and How to Prevent Osmotic Demyelination Syndrome 47:12 2024 FUTURES Steroid Use 1:21:52 Ask Me Anything: Traveling with Duchenne with Marissa Penrod, Adam Malone and Alan Chaulet 13:10 CAR T Cells: Beating Cancer with the Immune System 14:02 C15 A Longevity Game Changer? | The Studies Reviewed 34:46 2024 FUTURES Action is Therapy Getting Involved 02:23 Dream to Reality: Sarepta's ELEVIDYS Dosing at the CureDuchenne Clinic 1:21:29 Sauna Benefits Deep Dive and Optimal Use with Dr. Rhonda Patrick & MedCram 42:25 Navigating Duchenne treatment: AGAMREE & Catalyst Pathways 18:54 Methylation, the MTHFR gene, and your health. 43:58 Jon Fox's Trajectory Was Changed by a Second Opinion - It Takes Balls 30:11 2024 FUTURES - Clinical Trial Design, Lessons Learned and Implications for the Future Similar videos 01:28 Prosensa's Video Animation on Exon Skipping 32:40 Gene Editing for DMD 04:47 PORTRAIT OF DUCHENNE - Gene therapy and exon skipping 06:03 Advancing Care With Exon Skipping Therapies for Duchenne Muscular Dystrophy 47:59 In the Pipeline: Next-Gen Exon Skipping (PPMD's 2021 Virtual Annual Conference) 04:33 Efficacy Data for Eteplirsen for Skipping Exon 51 01:05 How Does It Work? | Antisense Oligonucleotide Therapy 58:11 An Insider’s View Into the Preclinical Development of an Approved Exon Skipping Drug 50:32 Annemieke Aartsma-Rus: Design of Exon Skipping Oligonucleotides 1:40:54 Restoring Dystrophin via Exon Skipping Strategies -- PPMD 2022 Annual Conference 02:54 Genes, Exons, and the Pathophysiology of DMD (Duchenne Muscular Dystrophy) 02:13 Fixing Genes with Antisense Oligonucleotides (ASOs) 02:01 Clinical Trials Antisense Oligonucleotides:Exon Skipping Efficiency In DMD Patient Samples 17:24 Alternative splicing ( mechanism and its regulation ) 01:38 RNA Splicing 08:23 Duchenne Muscular Dystrophy and Dystrophin 36:30 PepGen, Inc. Unlocking the Potential of Exon Skipping Therapeutics 40:33 Avidity: A New Approach to Exon Skipping 03:51 New Exon Skipping Drug in Clinical Development for Duchenne Muscular Dystrophy More results