Sep 16, 2021
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
25 minutes
Episodes
Ordered by publication date.
Sep 10, 2021
How a Familiar Face Can Lead to a Rare Disease Diagnosis
22 minutes
Sep 2, 2021
Bringing the Trial to the Patient
27 minutes
Aug 26, 2021
Targeting Rare and Chronic Kidney Diseases
23 minutes
Aug 19, 2021
Addressing the Delivery Challenges of Genetic Medicines
35 minutes
Aug 12, 2021
Targeting Tissues Throughout the Body with RNA Therapies
21 minutes
Aug 5, 2021
Delivering Rare Disease Therapies to Patients in Need
20 minutes
Jul 29, 2021
An Advocate's Journey through Diagnosis, Loss, and Hope
26 minutes
Jul 22, 2021
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
20 minutes
Jul 16, 2021
How a Small and Young Foundation Catalyzed Research into a Rare Disease
23 minutes
Jul 8, 2021
Overcoming the Limitations of Conventional Cell Engineering
30 minutes
Jul 2, 2021
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
20 minutes
Jun 24, 2021
Forging Gene Therapy Capacity and a Pipeline at the Same Time
18 minutes
Jun 18, 2021
Using A Natural DNA Repair Process to Improve Genetic Medicines
26 minutes
Jun 10, 2021
Empowering Rare Disease Patients with their Own Health Records
22 minutes
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