It Was All a Dream

Editor's Note: Patient Worthy is honored to share Michelle Patidar's patient story, provided to us by our friends at Elephants & Tea. To see the article in its original format,…

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First-in-Human Gene Therapy Offers Hope for Hunter Syndrome: Oliver’s Remarkable Turnaround

Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking early gains. Hunter syndrome is…

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The Things We Don’t Know

Editor's Note: We're honored to share this patient story-share by Jess Ippolito, originally published by our friends at Stork Genetics. To see the article in its original format, please click…

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Laura’s Battle Against Rickets

  Editor's Note: Patient Worthy is proud to share this story from GACI Global, originally published by the Italian Osservatorio Malattie Rare (OMaR). ARHR2 – Autosomal Recessive Hypophosphatemic Rickets Type…

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