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. ...
by Madaline Spencer | May 16, 2025
The U.S. Food and Drug Administration (FDA) approved Welireg (belzutifan) for patients ages 12 years and older with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma. Pheochromocytoma and paraganglioma are rare tumors that develop from...
by Madaline Spencer | May 16, 2025
Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, discusses how industry partners can support the mental health of rare disease communities. Dr. Freedman provides individual counseling, support group facilitation, and advises rare disease...
by Madaline Spencer | May 15, 2025
Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, provides an overview of urea cycle disorders (UCDs) and discusses developing novel therapies. UCDs are a group of rare, inherited metabolic disorders caused by deficiency of one of the six...
