The FDA has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy in children and adults

Within 24 hours of the first dose of Silmitasertib the patient showed significant clinical improvement and the oxygen requirement was weaned to room air. The patient was discharged from the hospital five days after starting Silmitasertib.

European Commission Approves Reblozyl (luspatercept) for the Treatment of Transfusion-Dependent Anemia in Adult Patients with Myelodysplastic Syndromes or Beta Thalassemia

The new drug program aims to decrease the amount of UTIs that would otherwise require antibiotic intervention.