Description: The Neglected Tropical Diseases are a group of conditions that affect the poorest one fourth of the world's ...

When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...

For many rare disease patients, access to expert care remains out of reach. Families often endure years of misdiagnoses and medical visits before finally receiving an accurate diagnosis. This is often ...

The selected projects demonstrated significant potential to address unmet needs within these rare communities. The recipients include: Fabry disease: Mitra Tavakoli, University of Exeter, Exeter, UK, ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

The Medicines and Healthcare products Regulatory Agency (MHRA) has announced plans to overhaul its approach to therapies for rare diseases. In a position paper, the agency highlighted that rare ...