by Peter Ciszewski | Nov 3, 2021
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research, discusses their study examining the prevalence and medical costs of some rare diseases. The study, published in the Orphanet Journal of Rare Diseases, is a retrospective study which...
by Peter Ciszewski | Nov 2, 2021
Sydney Stern, Director of Giant Cell Tumor Programs at the Life Raft Group and tenosynovial giant cell tumor (TGCT) patient, gives three pieces of advice to newly-diagnosed TGCT patients. TGCTs are a group of rare tumors that involve the synovium, bursae and...
by Peter Ciszewski | Nov 1, 2021
James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, discusses how neurofibromatosis type 1 (NF1) is diagnosed and its typical prognosis. NF1 is a genetic disorder due to mutations in the NF1 gene that leads to a plethora of symptoms...
by Peter Ciszewski | Oct 31, 2021
Results from a pivotal phase 3 clinical trial show dupilumab to be effective in treating a prurigo nodularis. The trial, known as the PRIME2 trial, examined the efficacy of dupilumab (n=78) to placebo (n=78) in patients with prurigo nodularis, a rare skin disorder....
by Peter Ciszewski | Oct 29, 2021
Jonathan Rigby, MD, President, Group CEO, and Board Member at Revolo Biotherapeutics, gives an overview of eosinophilic esophagitis (EoE). As Dr. Rigby explains, EoE is a chronic, allergic inflammatory disease that is characterized by the buildup of eosinophils...
