by Madaline Spencer | May 28, 2025
Kimberly LeBlanc, Genetic Counselor, Director of the Undiagnosed Diseases Network (UDN) Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases. The UDN is a NIH-funded...
by Madaline Spencer | May 26, 2025
Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases. The Children’s Tumor Foundation is an organization dedicated to developing treatments for patients with...
by Madaline Spencer | May 24, 2025
Alison Bateman-House, PhD, Assistant Professor Division of Medical Ethics at NYU Grossman School of Medicine, discusses ethical concerns in rare diseases and expanded access programs. Rare disease patients, caregivers, and healthcare practitioners often face...
by Madaline Spencer | May 23, 2025
Connie Lee, PsyD, Chief Executive Officer of Alliance to Cure Cavernous Malformations, discusses cerebral cavernous malformation (CMM) and the diversity of patient advocacy group initiatives. CMM is a rare, capillary-venous malformation characterized by...
by Madaline Spencer | May 21, 2025
Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, discusses the company’s current drug development programs. CAMP4 is a biopharmaceutical company focused on utilizing RNA to develop novel treatments that increase gene expression in patients...
