U.S. FDA Approves Biogen’s SPINRAZATM (nusinersen), The First Treatment for Spinal Muscular Atrophy
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چکیده
CAMBRIDGE, Mass.--(BUSINESS WIRE [2])--The U.S. Food and Drug Administrat ion (FDA) approved Biogen’s [3] (NASDAQ: BIIB) SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitat ing muscle weakness.
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U.S. FDA Approves Biogen’s SPINRAZATM (nusinersen), The First Treatment for Spinal Muscular Atrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE [2])--The U.S. Food and Drug Administrat ion (FDA) approved Biogen’s [3] (NASDAQ: BIIB) SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants and toddlers that is marked by ...
متن کاملU.S. FDA Approves Biogen’s SPINRAZATM (nusinersen), The First Treatment for Spinal Muscular Atrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE [2])--The U.S. Food and Drug Administrat ion (FDA) approved Biogen’s [3] (NASDAQ: BIIB) SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants and toddlers that is marked by ...
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Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, cannot compensate for the loss of SMN1 due to predominant skipping of exon 7. While various regulatory elements that modulate SMN2 exon 7 splicing have been proposed, intronic splicing...
متن کاملOverview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy
Spinal muscular atrophy (SMA) is a neurodegenerative disease primarily characterized by a loss of spinal motor neurons, leading to progressive paralysis and premature death in the most severe cases. SMA is caused by homozygous deletion of the survival motor neuron 1 (SMN1) gene, leading to low levels of SMN protein. However, a second SMN gene (SMN2) exists, which can be therapeutically targeted...
متن کاملIntrathecal Injections in Children With Spinal Muscular Atrophy
Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy. We summarize lumbar puncture experience in children with spinal muscular atrophy during a phase 1 open-label study of nusinersen and its extension. During the studies, 73 lumbar punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal ...
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تاریخ انتشار 2018