A groundbreaking achievement in genetic medicine has emerged as the European Medicines Agency (EMA) recommended approval of Waskyra (etuvetidigene autotemcel), the first gene therapy specifically designed to treat Wiskott-Aldrich syndrome…
Scientists have known for years that Wiskott-Aldrich syndrome (WAS) is caused by a gene on the X chromosome. The gene (the WASP gene) can be found throughout the immune…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In a story from Vietnam News, doctors in Hanoi, Vietnam at the Central Paediatrics Hospital announced for the first time that they had performed a successful bone marrow transplant using…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
According to a press release from the British-American biopharmaceutical company Orchard Therapeutics, the American Food and Drug Administration (FDA) has granted OTL-103, the Company's experimental Wiskott-Aldrich Syndrome drug, Regenerative Medicine…
Wiskott-Aldrich Syndrome Wiskott-Aldrich Syndrome (WAS) is a rare immune disorder which causes platelets in the blood to function abnormally. It also causes autoimmunity. Patients suffer from severe bleeding which can…
Austin Brown was diagnosed with Wiskott-Aldrich syndrome at three months of age. An uncommon blood disease, Wiskott-Aldrich syndrome results in a weakened immune system. Thanks to a stem-cell bone marrow…
Once upon a time there were three young brothers, and SPOILER ALERT- this part of the story, unfortunately, does not have a happy ending. Each brother suffered: Each had chronic…
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