The Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases

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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 [….]

Jeeva Informatics Hosts Rare Disease Panel on Decentralized, Patient-Focused Clinical Trials

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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.

10 Philadelphia Biotech Companies Targeting Rare Diseases

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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.

Rare Disease Challenge, REGENXBIO Tackles Duchenne Muscular Dystrophy

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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.