rare disease consortium

Introduction to the International Rare Diseases Research Consortium (IRDiRC)

3:26

Introduction to the International Rare Diseases Research Consortium (IRDiRC)
Rare diseases and the Bespoke Gene Therapy Consortium: Max Presa, Ph.D.

00:50

Rare diseases and the Bespoke Gene Therapy Consortium: Max Presa, Ph.D.
What's the impact of the Musketeers Memorandum on rare disease research?

2:11

What's the impact of the Musketeers Memorandum on rare disease research?
H3Africa Rare Disease WG: European Rare Disease Network (ERN): ITHACA

56:47

H3Africa Rare Disease WG: European Rare Disease Network (ERN): ITHACA
Longitudinal Study of Urea Cycle Disorders: Urea Cycle Disorders Consortium

6:11

Longitudinal Study of Urea Cycle Disorders: Urea Cycle Disorders Consortium
The Syneos Health Rare Disease Consortium

1:48

The Syneos Health Rare Disease Consortium
The Genetic Disorders Mucociliary Clearance Consortium

7:57

The Genetic Disorders Mucociliary Clearance Consortium
Genomics Research to Elucidate the Genetics of Rare Disease (GREGoR) Consortium Update - J. Posey

49:37

Genomics Research to Elucidate the Genetics of Rare Disease (GREGoR) Consortium Update - J. Posey
What is a Rare Disease?

2:33

What is a Rare Disease?
ARC Talks Webinar: Knowing Your Rights: Understanding Health insurance Coverage

51:33

ARC Talks Webinar: Knowing Your Rights: Understanding Health insurance Coverage
IRDiRC, Big Money, and Genetic Matchmaking

2:30

IRDiRC, Big Money, and Genetic Matchmaking
FDA Rare Disease Day 2023: “Intersections with Rare Diseases – A patient focused event”

7:32:56

FDA Rare Disease Day 2023: “Intersections with Rare Diseases – A patient focused event”
Developing customized, novel rare disease therapies: Meet Rich | The Disruptors

4:40

Developing customized, novel rare disease therapies: Meet Rich | The Disruptors
Rare Disease Research Day 2024

2:58:41

Rare Disease Research Day 2024
H3Africa Rare Disease WG Webinar

46:30

H3Africa Rare Disease WG Webinar
Register today for FDA's Rare Disease Day 2024

00:53

Register today for FDA's Rare Disease Day 2024
2021 PRO Consortium Workshop: Identifying COAs for Use in Rare Disease Treatment Trials

1:29:03

2021 PRO Consortium Workshop: Identifying COAs for Use in Rare Disease Treatment Trials
Rare Disease Diagnosis Patient Journey

2:18

Rare Disease Diagnosis Patient Journey
10 Tips for Newly Diagnosed Rare Disease Patients and Families

5:18

10 Tips for Newly Diagnosed Rare Disease Patients and Families
Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

10:59

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
This Young Man Is One Of Two People In The World Coping With A Rare, Nameless Disease | NBC News

9:42

This Young Man Is One Of Two People In The World Coping With A Rare, Nameless Disease | NBC News
Rare Disease Day Official Video 2024

1:01

Rare Disease Day Official Video 2024
Tips to Belong to the Rare Diseases Clinical Research Network (RDCRN)

2:33

Tips to Belong to the Rare Diseases Clinical Research Network (RDCRN)
Myasthenia Gravis and Vision

4:26

Myasthenia Gravis and Vision
Eight Rare Genetic Diseases Selected to Develop Gene Therapy “Development Playbooks”

1:53

Eight Rare Genetic Diseases Selected to Develop Gene Therapy “Development Playbooks”
About the Amyloidosis Research Consortium (ARC)

3:29

About the Amyloidosis Research Consortium (ARC)
Eosinophilic Esophagitis Explained

3:17

Eosinophilic Esophagitis Explained
What is a Rare Disease?

2:17

What is a Rare Disease?
What is Rare Disease Day?

1:37

What is Rare Disease Day?
Streamlining Gene Therapy Through the Bespoke Gene Therapy Consortium

6:03

Streamlining Gene Therapy Through the Bespoke Gene Therapy Consortium
Rare Disease Day Official Video 2023

00:20

Rare Disease Day Official Video 2023
Rare Disease Drug Development Education Series Trailer

1:50

Rare Disease Drug Development Education Series Trailer
Redefining Rare Disease Research - The Rare Disease Translational Center @jacksonlaboratory

7:33

Redefining Rare Disease Research - The Rare Disease Translational Center @jacksonlaboratory
Rare Disease Day 2018 Official Video

1:03

Rare Disease Day 2018 Official Video
The 6 Rare Diseases Changing Modern Medicine

12:12

The 6 Rare Diseases Changing Modern Medicine
National Rare Disease Week

00:59

National Rare Disease Week
Preparing for Rare Disease Day 2024

1:00:16

Preparing for Rare Disease Day 2024
H63D homozygous mutation syndrome explained: Wilson’s disease insidious iron sibling.

13:51

H63D homozygous mutation syndrome explained: Wilson’s disease insidious iron sibling.
Natural History Studies for Rare Diseases

57:24

Natural History Studies for Rare Diseases
Newborn Screening Dramatically Improves Survival Rates of Infants with SCID

10:25

Newborn Screening Dramatically Improves Survival Rates of Infants with SCID
Rare disease ages children eight times faster | 60 Minutes Australia

13:03

Rare disease ages children eight times faster | 60 Minutes Australia
10 Tips for New Rare Disease Advocates from Current Ones

2:36

10 Tips for New Rare Disease Advocates from Current Ones
Life with a Rare Genetic Disorder (SATB2)

3:08

Life with a Rare Genetic Disorder (SATB2)
What is a rare disease?

2:13

What is a rare disease?
Rare Disease Day 2015 Official Video

2:17

Rare Disease Day 2015 Official Video
Rare Disease Day, 29 Feb 2012 - Official Video

1:23

Rare Disease Day, 29 Feb 2012 - Official Video
Top 5 Rarest Diseases | House M.D.

8:40

Top 5 Rarest Diseases | House M.D.
The Exome Aggregation Consortium (ExAC) database: shedding light on human genetic variation

5:21

The Exome Aggregation Consortium (ExAC) database: shedding light on human genetic variation
Medical Mysteries and Rare Diseases: William Gahl at TEDxCMU 2011

17:05

Medical Mysteries and Rare Diseases: William Gahl at TEDxCMU 2011
Young Girl Cannot Feel Pain, Battles Rare Medical Condition CIPA | Good Morning America | ABC News

4:13

Young Girl Cannot Feel Pain, Battles Rare Medical Condition CIPA | Good Morning America | ABC News
National Rare Disease Policy 2021 explained - Health Sector Current Affairs - What is Rare Disease?

10:59

National Rare Disease Policy 2021 explained - Health Sector Current Affairs - What is Rare Disease?
Pre-Applicant Webinar for Multi-Omics for Health and Disease Consortium (2022)

1:30:25

Pre-Applicant Webinar for Multi-Omics for Health and Disease Consortium (2022)
World's Rarest Disease: The Human Mannequin | Documentary

13:57

World's Rarest Disease: The Human Mannequin | Documentary
Rare Disease Day 2018 official video

1:03

Rare Disease Day 2018 official video
Unexplainable Diseases | SERIOUSLY STRANGE #15

10:50

Unexplainable Diseases | SERIOUSLY STRANGE #15
Rare Disease Day Official Video 2024

1:01

Rare Disease Day Official Video 2024
David Adams - IRDiRC Interview Series | IRDiRC

1:04

David Adams - IRDiRC Interview Series | IRDiRC
Emerging Science in Kidney Disease with Pfizer

5:50

Emerging Science in Kidney Disease with Pfizer
Diseases I've Only Seen In Textbooks

17:11

Diseases I've Only Seen In Textbooks
Tip to Becoming a RDCRN Consortium – Find a Statistician

2:30

Tip to Becoming a RDCRN Consortium – Find a Statistician