Uploaded June 2026 | Updated September 2026, 2 weeks ago
A version of this video with audio description track is available here: youtube.com/watch?v=jvNsf4Dtvrc
Rare disease research is creating new paths for diagnosis, treatment, and broader medical discovery. Gene therapy can repair or replace faulty genes, and work on cystinosis has led to a stem cell platform now being applied to Danon disease, Sanfilippo syndrome C, Friedreich’s ataxia, and Alzheimer’s research. Funding programs support gene therapy, clinical trials, and new platform approaches for rare diseases. CAR-T cell research is also advancing treatment possibilities for pediatric brain tumors, including early results in children with DIPG and diffuse midline glioma. A patient advocate shares her daughter’s diagnostic odyssey and treatment for TUBB4A leukodystrophy. Together, these stories show why rare disease research matters beyond rarity. [7/2026] [Show ID: 41402]
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UCTV features the latest in health and medicine from University of California medical schools. Find the information you need on cancer, transplantation, obesity, disease and much more.
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(uctv.tv/science)
Science and technology continue to change our lives. University of California scientists are tackling the important questions like climate change, evolution, oceanography, neuroscience and the potential of stem cells.
UCTV is the broadcast and online media platform of the University of California, featuring programming from its ten campuses, three national labs and affiliated research institutions. UCTV explores a broad spectrum of subjects for a general audience, including science, health and medicine, public affairs, humanities, arts and music, business, education, and agriculture. Launched in January 2000, UCTV embraces the core missions of the University of California -- teaching, research, and public service – by providing quality, in-depth television far beyond the campus borders to inquisitive viewers around the world.
(https://www.uctv.tv)
A version of this video with audio description track is available here: youtube.com/watch?v=jvNsf4Dtvrc
Rare disease research is creating new paths for diagnosis, treatment, and broader medical discovery. Gene therapy can repair or replace faulty genes, and work on cystinosis has led to a stem cell platform now being applied to Danon disease, Sanfilippo syndrome C, Friedreich’s ataxia, and Alzheimer’s research. Funding programs support gene therapy, clinical trials, and new platform approaches for rare diseases. CAR-T cell research is also advancing treatment possibilities for pediatric brain tumors, including early results in children with DIPG and diffuse midline glioma. A patient advocate shares her daughter’s diagnostic odyssey and treatment for TUBB4A leukodystrophy. Together, these stories show why rare disease research matters beyond rarity. [7/2026] [Show ID: 41402]
Donate to UCTV to support informative & inspiring programming:
uctv.tv/donate
Please Note: Knowledge about health and medicine is constantly evolving. This information may become out of date.
More videos from: MIND - Medicine Informing Novel Discoveries
(uctv.tv/stem-cell/mind)
More from: Stem Cell Channel
(uctv.tv/stem-cell)
Explore More Health & Medicine on UCTV
(uctv.tv/health)
UCTV features the latest in health and medicine from University of California medical schools. Find the information you need on cancer, transplantation, obesity, disease and much more.
Explore More Science & Technology on UCTV
(uctv.tv/science)
Science and technology continue to change our lives. University of California scientists are tackling the important questions like climate change, evolution, oceanography, neuroscience and the potential of stem cells.
UCTV is the broadcast and online media platform of the University of California, featuring programming from its ten campuses, three national labs and affiliated research institutions. UCTV explores a broad spectrum of subjects for a general audience, including science, health and medicine, public affairs, humanities, arts and music, business, education, and agriculture. Launched in January 2000, UCTV embraces the core missions of the University of California -- teaching, research, and public service – by providing quality, in-depth television far beyond the campus borders to inquisitive viewers around the world.
(https://www.uctv.tv)
