The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
May 10th 2024
A Veterans Affairs study finds low alpha-1 antitrypsin phenotyping rates, indicating the need for improved healthcare provider education on AATD.
Vertex Initiates Second Phase 3 of Triple Combination for Cystic Fibrosis
March 1st 2018One week after Vertex Pharmaceuticals Incorporated initiated its first Phase 3 study of VX-659, tezacaftor and ivacaftor regimen for people with cystic fibrosis, the company has announced it is initiating a second.
NIH Awards $2.8 Million Grant to PhaseBio for Potential PAH Therapy
A $2.8 million Fast Track Small Business Innovation Research (SBIR) grant has been awarded to PhaseBio Pharmaceuticals from the National Institutes of Health (NIH) to support the clinical development of PB1046 in treating patients with pulmonary arterial hypertension (PAH).
Shire Advocates for Accurate, Timely Diagnosis of Patients on Rare Disease Day
On Rare Disease Day 2018, Shire plc will focus on promoting prompt diagnosis of rare diseases, as it is the most important issue affecting health, longevity and well-being for patients and their families..
Fortress and Aevitas Enter Sponsored Research Agreement with AAV Technology Expert
Fortress Biotech and its subsidiary Aevitas Therapeutics have entered into a sponsored research agreement with Dr. Guanping Gao’s laboratory at the University of Massachusetts Medical School.