The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with certain dystrophin gene mutations have a dystrophin protein that is more functional than their …
Reena Sharma, MD, discusses plans for a phase 3 clinical trial evaluating FLT201 in patients with Gaucher disease.
This Webinar examines what is PEGylation and the clinical uses of therapeutic proteins, and their applications in healthcare.
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.
