Children’s Tumor Foundation

  Annette Bakker, PhD, President and Chief Scientific Officer, of the Children’s Tumor Foundation discusses how her company works with the pharma and biotech industries, specifically SpringWorks Therapeutics. SpringWorks Therapeutics is planning to initiate...

Working With Achondroplasia Advocacy Groups

  Luca Santarelli, MD of Therachon discusses reaching out to achondroplasia advocacy groups. Achondroplasia is an autosomal dominant disorder characterized by dwarfism and disproportionate shortness of limbs, accompanied by life-altering complications....

FDA Approves Palynziq for PKU, a Rare Genetic Disease

The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU). Patients with PKU are born with an inability to break down phenylalanine (Phe), an amino acid present in...