Wait, How Do You Spell That? A Rare Disease Podcast
Health & Fitness
On today's episode, we sit down with two of the co-founders of GACI Global, a nonprofit organization centered around families affected by Generalized Arterial Calcification of Infancy. We also speak with the Vice President of Physician and Patient Strategies at Inozyme Pharma, which is pursuing novel therapeutics for the treatment of abnormal mineralization disorders such as GACI. Learn why close cooperation is so important in the development of treatments for rare conditions and why newborn screening is absolutely vital.
Important Links
GACI Global Homepage
GACI Global Worldwide Walk 2022
Inozyme Pharma Homepage
Inozyme Patient Resources
Inozyme Clinical Trial Information
Bridging the Challenges in Cell Therapies, feat. Dr. Brad Heller of Achieve Clinics
The Importance of Getting Involved, Feat. Friedreich‘s Ataxia Advocate Kyle Bryant
The Potential of CAR T-Cell Therapy, Feat. Dr. Robyn Stacy-Humphries
Not Just Surviving, But Thriving With Pheo vs. Fabulous
Hanging Onto Hope in the Face of AML
The Importance of Connection With Jordan‘s Guardian Angels
Awareness and Improvement: Discussing Narcolepsy With a Sleep Medicine Doctor
A Lifetime of Research with Dr. Cannon of the Periodic Paralysis Association
Building the Connections with the SYNGAP Research Fund
Making the "Invisible," Visible With Journalist Karina Sturm
Cure Mito Foundation: The Importance of Patient Registries
37 Years of Research With the TSC Alliance
Staying Strong and Pressing On With the Alagille Syndrome Alliance
Living Rare, Living Stronger: NORD Patient and Family Forum
Author Tom Seaman Talks About Adapting to Adversity
Sophie's Hope and GSD1B
Pemphigus and Pemphigoid: Talking Rare Disease Dermatology
The 2021RAREis Scholarship
Narcolepsy: A 20-Year Journey to Diagnosis
Sharing Patient Voices With Elephants and Tea
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