Канал: Cure Mito Foundation

Leigh syndrome patient registry

Leigh syndrome patient registry

4 Developing Diagnostic Resources for Leigh Syndrome Shamima Rahman PhD

4 Developing Diagnostic Resources for Leigh Syndrome Shamima Rahman PhD

1 Introduction Kasey Woleben

1 Introduction Kasey Woleben

2 Gene Therapy for Neurological Disorders Steven Gray PhD

2 Gene Therapy for Neurological Disorders Steven Gray PhD

3 Running for a Cure Greg MacCurtain

3 Running for a Cure Greg MacCurtain

5 Pluripotent Stem Cells and Brain Organoids for Leigh Syndrome Alessandro Prigione MD PhD

5 Pluripotent Stem Cells and Brain Organoids for Leigh Syndrome Alessandro Prigione MD PhD

7 The Immunological Origins of Leigh Syndrome Allison Hanaford PhD

7 The Immunological Origins of Leigh Syndrome Allison Hanaford PhD

8 Gene Therapy Development for SURF1 Related Leigh Syndrome Qinglan Ling PhD

8 Gene Therapy Development for SURF1 Related Leigh Syndrome Qinglan Ling PhD

9 Yeast Powered Drug Repurposing for Leigh Syndrome Ethan Perstein PhD

9 Yeast Powered Drug Repurposing for Leigh Syndrome Ethan Perstein PhD

10 Preventative Care and Medical Monitoring in Leigh Syndrome Sumit Parikh MD

10 Preventative Care and Medical Monitoring in Leigh Syndrome Sumit Parikh MD

11 Developmental and Rehabilitation Therapies Ibrahim Elsharkawi MD

11 Developmental and Rehabilitation Therapies Ibrahim Elsharkawi MD

12 Mito and Cannabis, What’s the Hype all About Sebastienne Cotte

12 Mito and Cannabis, What’s the Hype all About Sebastienne Cotte

13 Patient Registry and Data Panel Sophia Zilber, Pallavi Bakare, MS, Amanda Klein, PharmD CDCES,

13 Patient Registry and Data Panel Sophia Zilber, Pallavi Bakare, MS, Amanda Klein, PharmD CDCES,

14 Industry Panel Dr. Hansson, MD, PhD Dave Panake Dr.Tong, PhD Janice Keramedjian, Minyoung So

14 Industry Panel Dr. Hansson, MD, PhD Dave Panake Dr.Tong, PhD Janice Keramedjian, Minyoung So

15 Conclusion Jessica Fetterman, PhD, Kasey Woleben

15 Conclusion Jessica Fetterman, PhD, Kasey Woleben

Nora's story

Nora's story

Patient Registries Frequently Asked Questions

Patient Registries Frequently Asked Questions

Cure Mito Doodle Video

Cure Mito Doodle Video

Interoperability of Leigh Syndrome Registry Data with Regulatory Submission Standards

Interoperability of Leigh Syndrome Registry Data with Regulatory Submission Standards

Building a Worldwide Community: Leigh Syndrome Global Patient Registry

Building a Worldwide Community: Leigh Syndrome Global Patient Registry

Dr. Steven Gray - General Overview of Nervous System Targeted Gene Therapy

Dr. Steven Gray - General Overview of Nervous System Targeted Gene Therapy

Dr. Jessica Fetterman - Gaining Insights into Mitochondrial Diseases Using Stem Cells

Dr. Jessica Fetterman - Gaining Insights into Mitochondrial Diseases Using Stem Cells

Empower and Inspire 2022 Dr. Bellucci-Applying precision medicine discovery platform to SURF1 LS

Empower and Inspire 2022 Dr. Bellucci-Applying precision medicine discovery platform to SURF1 LS

Kasey Woleben, Dr Jessica Fetterman Wrap Up

Kasey Woleben, Dr Jessica Fetterman Wrap Up

Dr. Kramer Parenting Children with Mitochondrial Disease: Finding Space for Parental Well-Being

Dr. Kramer Parenting Children with Mitochondrial Disease: Finding Space for Parental Well-Being

Parent Panel

Parent Panel

Shannon O'Rourke SURF1 related Leigh Syndrome Health Data Insights

Shannon O'Rourke SURF1 related Leigh Syndrome Health Data Insights

Dr Alexandre Betourne - The Rare Diseases Cures Accelerator Data and Analytics Platform

Dr Alexandre Betourne - The Rare Diseases Cures Accelerator Data and Analytics Platform

Parag Shiralkar - Interoperability of LS Registry Data with Regulatory Submission Standards

Parag Shiralkar - Interoperability of LS Registry Data with Regulatory Submission Standards

Rhonda Facile - CDISC and How Data Standards Can Help Drive Development of Mito Treatments

Rhonda Facile - CDISC and How Data Standards Can Help Drive Development of Mito Treatments

Dr Weissig - Development of New Drugs for Mitochondrial Diseases with focus on Leigh Syndrome

Dr Weissig - Development of New Drugs for Mitochondrial Diseases with focus on Leigh Syndrome

Dr Matt Klein-Insights from over a decade of experience in mitochondrial disease drug development

Dr Matt Klein-Insights from over a decade of experience in mitochondrial disease drug development

Dr Anne Murphy Challenges in Drug Discovery for Mitochondrial Disorders

Dr Anne Murphy Challenges in Drug Discovery for Mitochondrial Disorders

Dr. Simon Johnson Immune Mediated Disease Pathogenesis in leigh Syndrome

Dr. Simon Johnson Immune Mediated Disease Pathogenesis in leigh Syndrome

Dr Prigione Patient-derived stem cells and brain organoids for modeling and drug discovery of LS

Dr Prigione Patient-derived stem cells and brain organoids for modeling and drug discovery of LS

Dr Michal Minczuk Mitochondrial Genome Engineering in Vivo

Dr Michal Minczuk Mitochondrial Genome Engineering in Vivo

Sophia Zilber Leigh Syndrome Global Patient Registry updates

Sophia Zilber Leigh Syndrome Global Patient Registry updates

Dr Saima Kayani Leigh Syndrome 101

Dr Saima Kayani Leigh Syndrome 101

Dr Vamsi Mootha - Keynote: Advances in Leigh Syndrome Research: Sterling Past, Golden Future

Dr Vamsi Mootha - Keynote: Advances in Leigh Syndrome Research: Sterling Past, Golden Future

Kasey Woleben - Introduction

Kasey Woleben - Introduction

Empower and Inspire - full conference

Empower and Inspire - full conference

Data Management and Analysis – Example from Leigh Disease Global Patient Registry

Data Management and Analysis – Example from Leigh Disease Global Patient Registry

Finding your family path - webinar with Blyth Lord, Courageous Parents Network

Finding your family path - webinar with Blyth Lord, Courageous Parents Network

The Mito Drug Development Journey

The Mito Drug Development Journey