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    Articles Tagged with "Rare Disease"

    Sindrome di Sanfilippo, la storia della piccola Ada: «Quella malattia non aveva nome». Cos'è la rara patologia genetica

    ilmessaggero.it • Medicine • Italy

    The article details the journey of a young girl, Ada, diagnosed with Sanfilippo syndrome type 3B, a rare genetic disorder, highlighting the challenges faced by her family and the importance of research and support.

    Sanfilippo syndrome rare disease genetic disorder child health Italy
    When Dad developed a worrying habit at 58, doctors put it down to stress. In fact it was the first sign of an unfathomably cruel disease | Daily Mail Online

    dailymail.co.uk • Medicine • UK

    A man's struggle with word-finding, initially attributed to stress, was later diagnosed as a rare form of dementia, highlighting the challenges of diagnosing and treating primary progressive aphasia.

    Dementia Aphasia Primary Progressive Aphasia rare disease Alzheimer's Research
    Gene-editing: Baby KJ was born with a rare, deadly disease. Doctors designed a fix – just for him

    smh.com.au • Medicine • World

    Doctors successfully used CRISPR gene editing to create a customized therapy for baby KJ, who suffers from a rare and deadly disease, offering hope for future treatments of similar conditions.

    Gene Editing CRISPR rare disease Gene Therapy Base Editing