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