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...
by Madaline Spencer | May 20, 2025
David Curren, patient advocate and board member for Breath of Hope Rhode Island, discusses his grandson’s diagnostic journey with idiopathic pulmonary hemosiderosis. Idiopathic pulmonary hemosiderosis (IPH) is a rare disease characterized by repeated...
by Madaline Spencer | May 19, 2025
Johanna Rossell, Senior Vice President and General Manager of Rare Diseases at Sumitomo Pharma America, discusses best practices for navigating challenges of the orphan drug market and provides personal experience through her work with congenital athymia. ...
