by Madaline Spencer | Jun 4, 2025
Jolan Walter, PhD, MD, Division Chief of Pediatric Allergy and Immunology at the University of South Florida, discusses genetic testing for WHIM syndrome. WHIM syndrome is an acronym for a rare immunodeficiency disorder: Each letter represents a...
by Madaline Spencer | Jun 2, 2025
Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). ALSP is a rare genetic, neurological condition largely due to damage to...
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...
