April 18, 2014 – Have you ever heard of Mucopolysaccharidosis Type IVA (Morquio A syndrome)? Most probably not.  And I would also think that only very few people are familiar with the “Rare Pediatric Disease Priority Review Voucher Program” by …

FDA approves Elosulfase Alfa [Vimizim] to treat the rare congenital enzyme disorder Mucopolysaccharidosis Type IVA (Morquio A syndrome) Read more »

March 31, 2014 –  The American Food and Drug Administration (FDA) is clarifying its previous recommendation related to prescribing Sidenafil [Revatio] for children with pulmonary arterial hypertension (PAH). Note that the pharmacologically active compound sildenafil is also the pharmacologically active …

Sildenafil [Revatio]: FDA Clarifies Warning About Pediatric Use for Pulmonary Arterial Hypertension (PAH) Read more »