Rare Disease Day at NIH 2024
Rare diseases affect an estimated 30 million people in the United States. On February 29, 2024, NIH will host its annual Rare Disease Day event to raise awareness about these disorders, the people they affect, and NIH collaborations that advance research for new treatments. Sponsored by the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center, the event will feature panel discussions, rare diseases stories, and more. Participants can share their thoughts, photos, and experiences via social media using the hashtag #RDDNIH.For more information go tohttps://ncats.nih.gov/rddAir date: 2/29/2...
Source: Videocast - All Events - September 25, 2023 Category: General Medicine Tags: Upcoming Events Source Type: video
Clinical Center GME Grand Rounds: Disease Discovery at the NIH Clinical Center: Translational Approaches to Rare Diseases
Clinical Center Grand Rounds
Graduate Medical Education Grand Rounds Disease Discovery at the NIH Clinical Center: Translational Approaches to Rare Diseases Steven Holland, MD Scientific Director, Division of Intramural Research NIH Distinguished Investigator Immunopathogenesis Section National Institute of Allergy and Infectious DiseasesFor more information go tohttps://clinicalcenter.nih.gov/about/news/grcurrent.htmlAir date: 7/26/2023 12:00:00 PM (Source: Videocast - All Events)
Source: Videocast - All Events - July 17, 2023 Category: General Medicine Tags: Upcoming Events Source Type: video
Rare Disease Day at NIH 2023
Rare diseases affect an estimated 30 million people in the United States. On February 28, 2023, NIH will host its annual Rare Disease Day event to raise awareness about these disorders, the people they affect, and NIH collaborations that advance research for new treatments. Sponsored by the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center, the event will feature panel discussions, rare diseases stories, and more. Participants can share their thoughts, photos, and experiences via social media using the hashtag #RDDNIH.
Explore virtual exhibits, view scientific posters, and network w...
Source: Videocast - All Events - October 12, 2022 Category: General Medicine Tags: Upcoming Events Source Type: video
Rare Disease Day at NIH 2022
Rare diseases affect an estimated 30 million people in the United States. On February 28, 2022, NIH will host its annual Rare Disease Day event to raise awareness about these disorders, the people they affect, and current NIH research collaborations underway. Sponsored by the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center, the event will feature interactive panel discussions, the sharing of rare stories, and more. Participants can share their thoughts, photos, and experiences via social media using the hashtag #RDDNIH. Explore virtual exhibits, view scientific posters, and network ...
Source: Videocast - All Events - November 16, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery (Day 3)
NIH invites stakeholders throughout the scientific research, advocacy, clinical practice, industry, and lay communities, including the general public, to attend the 3-day meeting and submit comments or questions relating to the effective, efficient and equitable distribution of gene-targeted therapies for individuals with rare diseases.
The goal of Day 3 is to discuss:• How will we ensure equitable access to gene-targeted therapies?For more information go tohttps://events-support.com/events/Gene-Targeted_Therapies_June_2021Air date: 6/17/2021 12:00:00 PM (Source: Videocast - All Events)
Source: Videocast - All Events - May 17, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery (Day 1)
NIH invites stakeholders throughout the scientific research, advocacy, clinical practice, industry, and lay communities, including the general public, to attend the 3-day meeting and submit comments or questions relating to the effective, efficient and equitable distribution of gene-targeted therapies for individuals with rare diseases.
The goal of Day 1 is to discuss:• Who are the individuals that could benefit from gene-targeted therapies – now and in the future?• What novel approaches are needed to enable development of gene-targeted therapies for all genetic rare diseases – now and in the future?• When is ...
Source: Videocast - All Events - May 17, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery (Day 2)
NIH invites stakeholders throughout the scientific research, advocacy, clinical practice, industry, and lay communities, including the general public, to attend the 3-day meeting and submit comments or questions relating to the effective, efficient and equitable distribution of gene-targeted therapies for individuals with rare diseases.
The goal of Day 2 is to discuss:• What infrastructure is required to disseminate gene-targeted therapies to individuals with rare diseases in need of treatment?For more information on the meeting and to view the agenda for all three days, visit:https://events-support.com/events/Gene-Targ...
Source: Videocast - All Events - May 17, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery [Day 3]
Currently, technological developments are offering the hope of new therapies for individuals with rare disease at a pace that has the potential for significantly challenging the existing infrastructure for efficient, effective, and equitable delivery. It is important therefore, to plan and be prepared for these challenges. Changes need to be implemented to improve the process of getting more treatments to more patients, more quickly. Gene-targeted therapies have the ability to provide treatments to multiple disorders rapidly. Gene-targeted therapeutics, such as virus-mediated gene replacement, somatic genome editing and ol...
Source: Videocast - All Events - April 19, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery [Day 2]
Currently, technological developments are offering the hope of new therapies for individuals with rare disease at a pace that has the potential for significantly challenging the existing infrastructure for efficient, effective, and equitable delivery. It is important therefore, to plan and be prepared for these challenges. Changes need to be implemented to improve the process of getting more treatments to more patients, more quickly. Gene-targeted therapies have the ability to provide treatments to multiple disorders rapidly. Gene-targeted therapeutics, such as virus-mediated gene replacement, somatic genome editing and ol...
Source: Videocast - All Events - April 19, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery [Day 1]
Currently, technological developments are offering the hope of new therapies for individuals with rare disease at a pace that has the potential for significantly challenging the existing infrastructure for efficient, effective, and equitable delivery. It is important therefore, to plan and be prepared for these challenges. Changes need to be implemented to improve the process of getting more treatments to more patients, more quickly.
Gene-targeted therapies have the ability to provide treatments to multiple disorders rapidly. Gene-targeted therapeutics, such as virus-mediated gene replacement, somatic genome editing and ...
Source: Videocast - All Events - April 19, 2021 Category: General Medicine Tags: Upcoming Events Source Type: video
Rare Disease Day at NIH 2021
Rare diseases affect an estimated 30 million people in the United States. On March 1, 2021, NIH will host its annual Rare Disease Day event to raise awareness about these disorders, the people they affect, and current NIH research collaborations underway. Sponsored by the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center, the event will feature interactive panel discussions, the sharing of rare stories, and more.
Participants can share their thoughts, photos, and experiences during the day via social media using the hashtag #RDDNIH. Virtual posters and exhibits and networking with e...
Source: Videocast - All Events - December 15, 2020 Category: General Medicine Tags: Upcoming Events Source Type: video
Importance of adding patient voice to healthcare innovation
Patient advocate Swapna Kakani shares her story of living with a rare disease and becoming a part of the emerging wave of patient leaders. (Source: Healthcare ITNews Videos)
Source: Healthcare ITNews Videos - October 26, 2020 Category: Information Technology Tags: Patient Engagement Quality and Safety Women In Health IT Source Type: video
Gaucher Disease: How a Rare Disease Provides a Window into Common Neurodegenerative Disorders
WALS Astute Clinician Lecture
The speaker, Dr. Ellen Sidransky, is the Branch Chief of the Medical Genetics Branch and is a pediatrician and geneticist in the National Human Genome Research Institute at National Institutes of Health (NIH). Her research interests include both clinical and basic aspects of Gaucher disease and Parkinson disease, studies of genotype/phenotype correlation and genetic modifiers, insights from mouse models, and novel treatment strategies. She played a lead role in establishing the association between glucocerebrosidase and parkinsonism. The author of over 200 publications, she continues to focus ...
Source: Videocast - All Events - October 20, 2020 Category: General Medicine Tags: Upcoming Events Source Type: video
Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies (Day 1)
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - January 22, 2020 Category: General Medicine Tags: Upcoming Events Source Type: video
Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies (Day 2)
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - January 22, 2020 Category: General Medicine Tags: Upcoming Events Source Type: video
