Orphan Drug Development, Pricing, and Access | CheckRare.com
Dennis Jackman, Senior Vice President, Public Affairs at CSL Behring discusses rare disease drug pricing, patient involvement in the drug development process, and drug access.
Dennis Jackman, Senior Vice President, Public Affairs at CSL Behring discusses rare disease drug pricing, patient involvement in the drug development process, and drug access.
Neuroblastoma is a rare childhood cancer characterized by a neuroendocrine tumor originating in neuroblasts or neural crest progenitor cells.
Barbara Burton, MD, discusses a recent study examining the effectiveness of idursulfase in young patients with MPS II.
AVP deficiency is a rare neuroendocrine disorder caused by impaired function of vasopressinergic neurons in the hypothalamus and posterior pituitary gland.
Three case studies exploring complexities of diagnosing, treating, and managing patients with various complement-mediated kidney disorders.
Erdheim-Chester disease is a rare condition affecting many parts of the body, it most commonly affects adults.
Jean Elwing discusses PAH research leading to better management of people with Pulmonary Arterial Hypertension
Anita D’Souza, MD, discusses results from the MajesTEC-2 and TRIMM-2 clinical trials for tec-DP in patients with multiple myeloma.
Drs. Aleena Banerji, Timothy Craig, and Marc Riedl, provide an overview of the discrepancies and discuss improving health equity in HAE.
Paula Ragan, X4 Pharmaceuticals, discusses the pathway of approval under the EMA for mavorixafor for patients with WHIM syndrome.
Dr Ozlem Goker-Alpan and Dr John Bach discuss best practices to manage pulmonary symptoms in lysosomal diseases