by CheckRare Staff | Jan 10, 2023
Mechanism of Action of SYNB1353 to Treat Homocystinuria Can Ficicioglu, MD, PhD, Clinical Director of the Metabolic Disease Program at Children’s Hospital of Philadelphia, discusses positive top-line data from the phase 1 clinical trial in...
by CheckRare Staff | Jan 9, 2023
What is Homocystinuria (HCU)? Can Ficicioglu, MD, PhD, Clinical Director of the Metabolic Disease Program at Children’s Hospital of Philadelphia, gives an overview of homocystinuria. As Dr. Ficicioglu explains, homocystinuria is a rare congenital...
by CheckRare Staff | Jan 7, 2023
Long-Term Use of Pegcetacoplan in Patients with PNH Christopher Patriquin, MD, Hematologist at Toronto General Hospital, discusses data from the long-term extension study of pegcetacoplan in adult patients with paroxysmal nocturnal hemoglobinuria (PNH)....
by CheckRare Staff | Jan 6, 2023
Christopher Patriquin, MD, Hematologist at Toronto General Hospital, discusses the diagnostic process for paroxysmal nocturnal hemoglobinuria (PNH), including symptoms that may suggest the disease. PNH is a rare, life-threatening blood disorder characterized by...
by CheckRare Staff | Dec 20, 2022
Lisa Borland, Vice President of Global Medical Affairs at Sarepta Therapeutics, discusses the clinical development program evaluating the safety and efficacy of SRP-9001, an investigational gene transfer therapy for Duchenne muscular dystrophy. Data from this...
