CHICAGO, Illinois – The American Society for Transplantation and Cellular Therapy® (ASTCT®) supports the decision by the U.S. Food and Drug Administration’s (FDA) to grant multiple myeloma patients earlier access to CAR T-cell therapy Abecma as a second-line treatment and the CAR T therapy Carvykti as a third-line treatment. The...
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BOSTON, Mass. — The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today announced that it is petitioning the U.S. Food and Drug Administration (FDA) to review the New Drug...
Boston — The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, recognizes the first annual Barth Syndrome Awareness Day. Barth syndrome is an ultra-rare, life-threatening, genetic disease primarily affecting males....
Boston — The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, recognizes the first annual Barth Syndrome Awareness Day. Barth syndrome is an ultra-rare, life-threatening, genetic disease primarily affecting males....
Washington, DC – Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center for Biologics...
Many people with Lambert-Eaton myasthenic syndrome (LEMS) wait an extremely long time for a diagnosis. Our family waited nine months, a relatively short time compared with others in the LEMS community. As we waited for a diagnosis to be confirmed for our 16-year-old daughter Grace, a fog of uncertainty hung over our...
NORTH BETHESDA, Md. — The Foundation for the National Institutes of Health (FNIH) announces the launch of a public-private partnership to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development...
Toronto, Ontario – Using a novel methodology, researchers at The Hospital for Sick Children (SickKids) are the first in paediatric research to use data from an international real-world cohort to overcome the barriers associated with conducting randomized clinical trials in children with rare diseases. The gold standard for evaluating new...
BAR HARBOR, Maine – The Jackson Laboratory, an independent, nonprofit biomedical research institution, announced the appointment of Lon Cardon, Ph.D., FMedSci, a pioneer in human genetics and drug discovery, as its next president and chief executive officer. Effective on November 29, Cardon will succeed current President and CEO Edison Liu...
WASHINGTON, DC — The Melanoma Research Alliance (MRA), the largest non-profit funder of melanoma research worldwide, welcomes the U.S. Food and Drug Administration (FDA) decision to approve Iovance Biotherapeutics’ AMTAGVI™ (lifileucel) for the treatment of patients with advanced melanoma in the second line treatment setting. AMTAGVI is the first individualized...