Grant compassionate use of experimental therapy for a life-threatening childhood disease
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Our 2 year old daughter, Radha Rangarajan, was diagnosed with late-infantile metachromatic leukodystrophy on April 13th, 2018. Without treatment, Radha will experience a rapid neurological decline over the next couple of months resulting in incapacitation, leaving her non-verbal, blind, and prone to seizures.
The disease then plateaus leaving her in a vegetative state for several years, with most children not surviving past their 8th birthday. There are no available treatment options and Radha is not eligible for any clinical trials.
Shire Pharmaceuticals has developed an enzyme replacement therapy, SHP611 (also known as HGT-1110), which has undergone early testing with promising results. Our requests for compassionate use of the medication have been denied by Shire despite this being her only chance at life.
Radha's condition continues to worsen every day and by the time Shire's proposed follow up trial begins enrolling patients it will be too late for her as the disease will have progressed too far.
We respectfully request that Shire Pharmaceuticals grants compassionate use of this enzyme therapy for our sweet little girl. Her life depends on it. Please sign this petition to show your support for compassionate use of SHP611/HGT-1110 for Radha Rangarajan. Thank you.
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