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...
by CheckRare Staff | Dec 15, 2022
Can Ficicioglu, MD, PhD, Clinical Director of the Metabolic Disease Program at Children’s Hospital of Philadelphia (CHOP), discusses the management of homocystinuria. Homocystinuria is a rare congenital metabolic disease characterized by extremely...
by CheckRare Staff | Dec 14, 2022
Ajai Chari, MD, Professor of Medicine and Director of Clinical Research, Multiple Myeloma Program, Mount Sinai School of Medicine, discusses results from the phase 1/2 MonumenTAL-1 clinical trial. These results were recently presented at this year’s American...
