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Hemophilia

Hemophilia

Latest Clinical Trials Rare Care Podcast The Pursuit of Redosable Gene Therapies for Hemophilia with Dr. Doug Kerr

Pompe Disease

Pompe Disease

Latest Clinical Trials Pompe Disease Clinical Insights Rare Care Podcast Barry Byrne, MD, director of the University of Florida’s Powell Gene Therapy Center in Gainesville, has spent years researching potential gene therapies for Pompe disease. In this edition of the Rare Care podcast, Dr. Byrne comments on the FDA’s recent approval of avalglucosidase alfa-ngpt (Nexviazyme)…

Long Chain Fatty Acid Oxidation Disorder News

Long Chain Fatty Acid Oxidation Disorder

Latest Clinical Trials Rare Care Podcast Perspectives on Long Chain Fatty Oxidation Disorder With Dr. Jerry Vockley  Ultragenyx CMO Dr. Camille Bedrosian Discusses the First FDA-Approved Treatment for LCFAOD”

Lysosomal Acid Lipase Deficiency News

Lysosomal Acid Lipase Deficiency

Latest Clinical Trials Rare Care Podcast An Update on LAL-D With Dr. Paul Martin of Nationwide Children’s Hospital

GIST news

Gastrointestinal Stromal Tumor

Latest Clinical Trials GIST Clinical Insights Rare Care Podcast Advocating for People With Gastrointestinal Stromal Tumor: An interview with Peter Knox, senior research director at the Life Raft Group