Happy Rare Disease Week!
This week has been all about rare diseases. Today, we’re highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings of NICE. After that, we have updates on trials for patients with proteus syndrome and hypoparathyroidism.
Sit back and enjoy this week’s Editor’s Choice.
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Laura shares a story about a condition that affects her family.
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Report Explains How NICE Policies are Failing Rare Disease Patients
NICE is responsible for coming to the aid of rare patients in the UK, but not everyone is happy with how inaccessible medication can be.
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The Yet Another Completed Study Supports Miransertib as a Therapy for Proteus Syndrome
A therapy should, ideally, have multiple studies that show its value. A new study reinforced the merit of a treatment for proteus syndrome.
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International Phase 2 Clinical Trial Initiated for New Potential Hypoparathyroidism Therapy
Check out PaTH Forward, a global trial bringing home to the HP community!
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