NICE the drug decision making body have announced their final decision today, Wednesday 15 February 2017, not to recommend Kanuma (Sebelipase alfa), the treatment for individuals suffering from Lysosomal Acid Lipase Deficiency (LAL D). Kanuma was given fast track approval
The U.S. Food and Drug Administration have permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns. The Seeker system is designed to detect Mucopolysaccharidosis Type I (MPS I), Pompe, Gaucher and Fabry.
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ABO-102, the leading clinical gene therapy program for Sanfilippo syndrome type A patients, has demonstrated central nervous system (CNS) and peripheral organ disease biopotency First high dose cohort patient is enrolled, and all patients (n = 4) have cumulative 644
individual and families
supported in 2015
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