by Madaline Spencer | Sep 24, 2025
Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses long-term safety and efficacy data on givinostat for patients with Duchenne muscular dystrophy (DMD). DMD is a rare neuromuscular disorder characterized by progressive...
by Madaline Spencer | Sep 23, 2025
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide HCI) for the treatment of patients with Barth syndrome. The treatment is indicated to improve muscle strength in adult and pediatric patients weighing at least 30...
by Madaline Spencer | Sep 23, 2025
Greg Palko, Vice President and Oncology Franchise Head of Kyowa Kirin North America, discusses a new cutaneous T-cell lymphoma (CTCL) Staging Tool. CTCL is a rare group of malignancies that attack the body’s immune lymphatic system, affecting both...
by Madaline Spencer | Sep 22, 2025
Michele Rayes, Associate Director of the HypoPARAthyroidism Association and patient, discusses the diagnosis and management of hypoparathyroidism. Hypoparathyroidism is a rare endocrine disorder in which the parathyroid glands do not produce enough parathyroid...
by Madaline Spencer | Sep 19, 2025
Sharon Halperin, Research Director for Lipodystrophy United and patient, discusses her diagnostic journey with lipodystrophy. Lipodystrophies are rare metabolic disorders characterized by the loss of adipose tissue. This leads to complications such as...
