Hemophilia A Treatment Options | CheckRare.com
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.
Mario Maldonado, MD, Head of Clinical Development for Global Endocrinology at Recordati Rare Diseases, discusses current treatment options for patients with Cushing’s syndrome.
Dr Ozlem Goker-Alpan and Dr John Bach discuss best practices to manage pulmonary symptoms in lysosomal diseases
Diane Schriner, Vice President of Erdheim-Chester Disease Global Alliance, provides an overview of Erdheim-Chester disease (ECD).
Learn the role of protein activators in lysosomal disorders and the role of inflammatory pathways in sphingolipid disorders.
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
Stiff person syndrome (SPS) is a rare, progressive disease that affects the nervous system, specifically the brain and spinal cord.
Pisit “Duke” Pitukcheewanont, MD, Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 for treatment of growth hormone deficiency.
Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments.
Jula Inrig, MD, discusses a clinical trial that tested sparsentan for treatment of IgA nephropathy, compared to traditional RAS inhibitors.
Kurt Gunter, MD, Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at ASBMR 2024.