Without Xenpozyme, Caitlin Monachan’s rare genetic disorder may kill her. But she must wait for the medication to be approved in the UK
Without Xenpozyme, Caitlin Monachan’s rare genetic disorder may kill her. But she must wait for the medication to be approved in the UK
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Mary Catchpole, 19, receives ‘life-changing’ treatment for immune condition after NHS approval
A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.
Family of two-year-old face the ultimate ‘race against time’ as they fight to access life-saving treatment before it is too late
Stephanie Eva, 63, says the ‘postcode lottery’ of treatment for motor neurone disease make her feel like her life doesn’t matter
Huw Jones died in February after suffering from bile duct cancer. His family believe that without zanidatamab, they would not…
Medicines watchdog approves two treatments for patients with spinal muscular atrophy
