by Madaline Spencer | Jul 29, 2024
The U.S. Food and Drug Administration (FDA) has approved Brineura (cerliponase alfa) for the treatment of Neuronal ceroid lipofuscinosis 2 (CLN2) disease in children under three years of age. CLN2 disease is a rare disorder that affects the nervous...
by Madaline Spencer | Jul 29, 2024
Patty Ketting, Executive Director of the HypoPARAthyroidism Association and a patient with hypoparathyroidism, discusses challenges of hypoparathyroidism. Hypoparathyroidism is is a rare endocrine disorder in which the parathyroid glands in the neck do...
by Madaline Spencer | Jul 26, 2024
Kristina Allikmets, MD, PhD, Senior Vice President, Head of R&D for the Plasma-Derived Therapies Business Unit at Takeda, discusses a breakthrough therapy for the treatment of CIDP. Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare...
by Madaline Spencer | Jul 25, 2024
Maria Fleseriu, MD, Director of the Pituitary Center at Oregon Health and Science University, discusses clinical trials testing osilodrostat for the treatment of Cushing’s disease. Cushing’s disease is a rare condition characterized by elevated levels of...
by Madaline Spencer | Jul 24, 2024
Michelle Reyes, Associate Director of the HypoPARAthyroidism Association and patient with the disease, discusses living with hypoparathyroidism and how the HypoPARAthyroidism Association can help. Hypoparathyroidism is is a rare endocrine disorder in...
