Patients & Caregivers
Outcome Measures for Batten Disease
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CEO of Abeona Discusses Gene Therapy for Sanfilippo Syndrome
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Protalix's Plant Derived Fabry ERT in Phase 3 Study
What is the Best Cognitive Test for Neurodegenerative Diseases?
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Dear FDA and Pharma - Your Risk Benefit Ratio is Not My Risk Benefit Ratio
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Newborn Screening for Pompe Disease - A Catalyst For Understanding a Rare Disease
Shire's Rare Disease Products
The Hidden Costs of Managing a Rare Disease
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