17 minutes |
Jun 8, 2023
Bringing Regenerative Medicine to a Rare Bone Condition in Children
27 minutes |
Jun 1, 2023
Bringing Precision to CRISPR-Based Genome Editing
19 minutes |
May 25, 2023
Capturing Patients’ Experience in Their Daily Lives
20 minutes |
May 18, 2023
From One to Many
22 minutes |
May 11, 2023
A Nasal Spray to Curb Excessive Eating in Prader-Willi
26 minutes |
May 4, 2023
How One Foundation Is Laying the Groundwork to Advance Treatments for an Ultra-Rare Disease
29 minutes |
Apr 27, 2023
Forging a New Path for Medicine
42 minutes |
Apr 20, 2023
Restoring Hearing through Gene Therapies
35 minutes |
Apr 13, 2023
Creating a Sustainable Reimbursement Model for Ultra-Rare Therapies
18 minutes |
Apr 5, 2023
Lumos Hopes Its Oral Alternative to HGH Will Fuel Its Growth
20 minutes |
Mar 30, 2023
Advancing a Gene Therapy for a Rare and Fatal CNS Disorder
26 minutes |
Mar 23, 2023
Addressing the Barriers to Patient Participation in Clinical Trials
27 minutes |
Mar 16, 2023
A Venture Philanthropist Makes the Case for Advocates Taking Equity
45 minutes |
Mar 9, 2023
Seeking a Sustainable Business Developing on N-of-1 Therapies
16 minutes |
Mar 3, 2023
How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition
20 minutes |
Mar 3, 2023
A Next-Generation RNA Therapy Targets Telomere Disorders
29 minutes |
Feb 24, 2023
BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families
37 minutes |
Feb 16, 2023
Translating Urgency into Science
33 minutes |
Feb 10, 2023
Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments
20 minutes |
Feb 2, 2023
Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X