by Madaline Spencer | May 30, 2025
The U.S. Food and Drug Administration (FDA) has approved Khindivi (hydrocortisone) oral solution as a replacement therapy in patients five years of age and older with adrenocortical insufficiency. Adrenocortical insufficiency is an endocrine disorder characterized by...
by Madaline Spencer | May 30, 2025
Melanie Lendnal, Senior Vice President of Policy & Advocacy at The ALS Association, discusses the Accelerated Approval of Qalsody (tofersen) for patients with amyotrophic lateral sclerosis (ALS). ALS is a rare motor neuron disease leading to problems...
by Madaline Spencer | May 29, 2025
The U.S. Food and Drug Administration (FDA) has approved Yutrepia (treprostinil) inhalation powder for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease. PAH PAH is a rare condition affecting the...
by Madaline Spencer | May 29, 2025
James Griffin, sickle cell anemia patient advocate, gives advice to patients with sickle cell anemia and healthcare providers. Sickle cell anemia is a rare genetic disease in which the body produces red blood cells that are sickle-shaped. The sickle...
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...
