Rare Diseases

RegulatoryJuly 22, 2026

Long-Term Safety Follow-Up for Seattle Children's Gene Therapy Products Initiated

Seattle Children's Hospital is conducting a long-term follow-up study to assess the safety of its gene therapy products over a period of up to 15 years.

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NIH Undiagnosed Diseases Network Aims to Enhance Diagnosis of Rare Conditions

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Long-Term Safety Follow-Up for Seattle Children's Gene Therapy Products Initiated

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MHRA Launches Consultation for Earlier Licensing of Rare Disease Therapies

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Efzimfotase alfa shows positive Phase III results in hypophosphatasia

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