FDA approves Brineura for type of Batten disease – Healio
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FDA approves Brineura for type of Batten disease
Healio The FDA has approved cerliponase alfa to curb the loss of ambulation among patients aged 3 years and older diagnosed with late infantile neuronal ceroid lipofuscinosis type 2, an extremely rare and fatal autosomal recessive neurodegenerative disorder, … New drug for rare disease wins approval, a “comfort” for family too late to benefit FDA action buoys Memphis family battling brain disease BioMarin’s Brineura Gets FDA Approval As First Treatment For Type Of Batten Disease |
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