Rare diseases impact populations across the globe, but despite that wide swath of known patients, the majority of clinical work is conducted in the United States and the European Union. There is a lack of diversity within the patient populations being tested for potential therapies, including cutting-edge gene therapies.
Leading up to Rare Disease Day on Monday, February 28, the National Organization for Rare Disorders (NORD) is highlighting the partners and supporters of this incredible international awareness day, sharing their reasons and motivations being participation, advocacy, education and engagement of the general public around rare diseases.
The Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases – A Five-year Commitment From Takeda Will Help Children’s National Launch a [….]
The National Economic Burden of Rare Disease Study conducted on behalf of the EveryLife Foundation for Rare Diseases took a first-of-its-kind look at the economic burden of rare diseases in the United States for the year 2019.
In recognition of Rare Disease Day 2021, Jeeva Informatics (Jeeva) brought together a panel of experts to discuss the importance of transforming the clinical trial process for rare diseases. The panel included Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases; Thomas Lester, Former Executive Director, Audentes Therapeutics; Scott Schliebner, Senior Vice President, Scientific Affairs & Therapeutic Expertise, PRA Health Sciences; and Dr. Eric Sid, Program Director, National Center for Advancing Translational Science, Office of Rare Disease Research, (NCATS)/NIH.
Philadelphia, the birthplace of cell and gene therapy, is home to a number of companies focused on developing multiple treatment options for rare diseases, those that affect less than 200,000 people in the United States.
Duchenne Muscular Dystrophy is a severe degenerative muscle disease that impacts about one in 5,000 boys per year across the globe. The disease causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down.