In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
In 1956, Dr. C. Miller Fisher first described Miller Fisher syndrome after seeing three patients who presented with absent tendon reflexes, ataxia (impaired coordination), and eye muscle weakness. Eventually,…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to diagnose, prevent, or treat rare conditions (those affecting fewer than 200,000 people in…
The 1980 World Series was a grand event for the Philadelphia Phillies. It was the last inning, and the Phillies were up by 3. With two players out and…
The Muscular Dystrophy Association (MDA) held its MDA Clinical & Scientific Conference from March 19-22, 2023 to share research, cutting-edge medical advancements, and clinical care practices within the muscular…
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…
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