US Edition‘Life-changing’ drug for teenager who lost mother, aunt and uncle to condition
- A teenager named Mary Catchpole has become the first person in Europe to receive a life-changing drug approved for use on the NHS.
- Mary Catchpole suffers from activated PI3-Kinase delta syndrome, which significantly weakens the immune system.
- The drug, leniolisib , is the first targeted treatment for this condition and allows for easier management compared to previous medications.
- Experts believe this treatment represents a major advancement in medical care for rare genetic disorders.
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khbrknews'Life-changing' drug for teenager who lost mother to genetic condition
The new drug called Lleniolisib (Joenja) is a simple tablet taken twice a day and is the first ever targeted treatment for activated PI3-Kinase delta syndrome (APDS), a rare inherited disorder.
·United Kingdom
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Patient with debilitating inherited condition receives new approved treatment on the NHS in Europe first
26.06.2025 - A teenager who has lost family members including her mother because of a rare genetic hereditary illness has become the first patient in the UK and Europe to have a new treatment developed by Cambridge researchers and approved for use on the NHS.
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