by Madaline Spencer | Oct 6, 2026
The US Food and Drug Administration (FDA) has approved Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in patients weighing 30 kg (66 lbs) or more. oHCM is a rare genetic...
by Madaline Spencer and Ailene Cantelmi | Oct 5, 2026
Paz Vellanki, MD, PhD, Vice President of Clinical Development at Precision for Medicine, discusses the FDA CDER’s Accelerating Rare Disease Cures (ARC) Program. The ARC Program was launched in 2022 with the goal of accelerating the development of safe and...
by Madaline Spencer | Oct 1, 2026
The US Food and Drug Administration (FDA) has approved Gazyva (obinutuzumab) to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid dependent childhood-onset idiopathic nephrotic syndrome (INS) who...
by Madaline Spencer | Sep 30, 2026
The US Food and Drug Administration (FDA) has approved Emcitate (tiratricol) for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency. MCT8 deficiency is a rare disorder of brain development that causes moderate...
by Madaline Spencer and Ailene Cantelmi | Sep 30, 2026
A study published in The Lancet evaluated the safety and efficacy of ralinepag in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the heart and lungs, characterized by abnormally high blood pressure (hypertension) in the...
