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.
About a year ago, BioBuzz attended the grand opening of Vigene Biosciences (Vigene) brand new headquarters in Rockville, Maryland. It was a fantastic day for Vigene, the BioHealth Capital Region (BHCR), and for the patients and families of people suffering from cancer and rare genetic diseases.
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.
Moustapha El-Amine, Ph.D. Joins Insmed Incorporated as Executive Director of Global Search and Evaluation (S&E)
Why Batten Hope Foundation is Funding Gene Therapy Clinical Trials for Children with Rare Diseases To hear Gina Hann speak about her son Joseph’s battle with an ultra-rare form of [….]
Five Biotechs Poised to Advance Their Rare Disease Programs [2022 updated] On the cusp of rare disease day, we’re checking in with Maryland biotech companies at the forefront of rare [….]
Cerecor Announces $43 Million Deal with AYTU BioScience to Sell Pediatric Portfolio and Advance Towards NDA
