Formed 15 years ago, the Genetic Disorders of Mucociliary Clearance Consortium is now co-led by UNC’s Stephanie Davis, MD, to continue advancing knowledge and treatments for genetic chronic pulmonary conditions. Source UNC Health Care To continue the push toward clinical trial readiness for individuals with rare diseases, the National Center for Advancing Translational Sciences, in
Patients with neurological disease that causes blindness, disability go treatment free Source Northwestern University A stem cell transplant reversed a debilitating neurological disease that causes half of the patients to go blind and lose the ability to walk five years after diagnosis. Most of the patients stayed better five years after the transplant and were
The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s potential therapeutic activity. Suvodirsen already had been granted orphan drug designation for the treatment of DMD
Reneo Pharmaceuticals, a clinical stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company’s lead drug candidate, REN001, for the treatment of fatty acid oxidation disorders (FAOD). “There is a pressing need for effective treatments for patients suffering from
The authors outline the latest discoveries in the biology of myelofibrosis (MF), discuss current clinical management of patients with MF, and summarize the ongoing clinical trials that hope to change the landscape of MF treatment. Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by ineffective clonal hematopoiesis, splenomegaly, bone marrow fibrosis, and the propensity for transformation
The collaborative project between the organizations will kick off at a launch meeting in September and will aim to reduce barriers for the development of new treatments and cures for rare diseases The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD
