01:41

The Greatest Gift: Jamal Hill

04:05

The Greatest Gift: Lily Sander

03:36

The Greatest Gift: Gary Berg

03:55

The Greatest Gift: Allison Taylor

02:48

The Greatest Gift: Rob Prior

38:40

You Are UNSTOPPABLE

12:27

Development of Gene Therapy for Rare Diseases

05:34

Clinical Development of CLC-1 Inhibitors For the Treatment of CMT

10:03

You and the Future of Clinical Trials in CMT

14:47

A Guide to Clinical Research: One Family's Journey

49:59

Fueling Progress With ENDGAME - The Campaign to End CMT1A

20:55

Lessons Learned From SOD1 ALS Clinical Development: Opportunities for CMT Drug Development

14:18

The State of Drug Development for CMT

04:56

Living With CMT

43:32

What is Happening to my Nerves and Body

04:58

Living With CMT

14:54

UNSTOPPABLE

14:09

Opening/Patients Taking Action

06:53

Patient Voices: How Your Scientific Progress Provides Hope

08:35

Patient Voices - Living With CMT

17:13

TRPV4 Inhibition in the Treatment of CMT2C

22:09

Supporting Translation of CMT1A Gene Therapy Through AAV-mediated Silencing of PMP22

17:09

Development of Gene Replacement Therapy for CMT4A Demyelinating Neuropathy

22:06

The Development of Tissue Tropic AAVs – Considerations Inside Out

11:59

Developing Lipid Nanoparticles for Non-Liver RNA Delivery

20:26

Current Status of Clinical Outcome Assessments and Biomarkers in CMT

44:36

Understanding the Criteria For Pursuing a Drug Program for CMT

20:08

Discovery of Small Molecules that Alter the Trafficking of PMP22 as A Potential Route

18:14

Developing Best-in-class HDAC6 Inhibitors for the Treatment of CMT1A

17:50

FDA Efforts to Advance Medical Product Development for Rare Diseases

17:16

Govorestat for CMT-SORD: Designing a Clinical Trial for a Slow Progressing CMT

20:39

Understanding Basic Biological Mechanisms to Identify Therapeutic Targets in CMT

20:50

Clinical Development of CLC- Inhibitor for the Treatment of Charcot-Marie-Tooth Disease

30:25

Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 3

45:57

Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 2

34:54

Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 1

01:13:23

CMT1A Clinical Trial and Drug Development Update

03:44

Peter DeSilva - CMT Story

01:13:55

CMT1A Research Update: Looking Towards Clinical Trials and Treatments

00:23

The Greatest Gift: Bentzin $500,000 Match

00:29

The Greatest Gift: Eloise Schlafly

00:44

The Greatest Gift: Tara Haik

00:27

The Greatest Gift: Bentzin $500,000 Match

00:33

The Greatest Gift: Michelle Moon

00:34

The Greatest Gift: Lily Sander

01:21

The Bentzin Family: A $500,000 Match

53:20

Pushing Boundaries in CMT Research Early Stage Biotech Companies' Evolution and Insights on Therapy

44:52

Translating Academic Science to Business

59:16

Clinical Trial Readiness

26:59

The State of Gene Therapy in CMT

19:54

Models of CMT

01:20:18

Common Mechanisms of Disease

08:06

Challenges in Drug Development

19:30

Overview of the CMT Drug Development Pipeline

36:46

Patient Fireside Chat - Life with CMT

50:58

Keynote Address

36:35

Becoming Part of the Solution

01:01:38

Patient Powered Perspectives Biotech Insights on CMT and Collaborating with Patients

45:24

Understanding the Importance of Genetic Testing

29:10

Patient Voices