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Challenges and opportunities in clinical trials for spinal muscular atrophy

Challenges and opportunities in clinical trials for spinal muscular atrophy Views & Reviews Challenges and opportunities in clinical trials for spinal muscular atrophy D. Hirtz, MD; S. Iannaccone, MD, FAAN; J. Heemskerk, PhD; K. Gwinn-Hardy, MD; R. Moxley III, MD; and L.P. Rowland, MD Abstract—Spinal muscular atrophy (SMA) is the most common fatal neuromuscular disease of infancy. SMA type I is the most severe and mortality is usually due to respiratory failure. In type II the disability is of later onset and less severe, and prognosis has improved primarily due to supportive care. Type III is the mildest form with onset usually of weakness in adolescence or young adulthood. SMA is an autosomal recessive disorder with deletions or mutations of the gene at the 5 q11 locus. There is no specific prevention or treatment, but current progress toward potential therapies has been substantial and several candidates including histone deacetylase (HDAC) inhibitors are under consideration for further evaluation. The authors sought to address the challenges and opportunities for testing new therapies for SMA. NEUROLOGY 2005;65:1352–1357 Spinal muscular atrophy (SMA) is a severe and often and profound hypotonia are noticeable in the first devastating neurologic disorder of infants and chil- few months of life. There is a striking discrepancy dren. The http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Neurology Wolters Kluwer Health

Challenges and opportunities in clinical trials for spinal muscular atrophy

Rowland, L P.
Neurology , Volume 65 (9) – Nov 1, 2005
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References (36)

ISSN
0028-3878
eISSN
1526-632X
DOI
10.1212/01.wnl.0000183282.10946.c7
pmid
16275820
Publisher site
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Abstract

Views & Reviews Challenges and opportunities in clinical trials for spinal muscular atrophy D. Hirtz, MD; S. Iannaccone, MD, FAAN; J. Heemskerk, PhD; K. Gwinn-Hardy, MD; R. Moxley III, MD; and L.P. Rowland, MD Abstract—Spinal muscular atrophy (SMA) is the most common fatal neuromuscular disease of infancy. SMA type I is the most severe and mortality is usually due to respiratory failure. In type II the disability is of later onset and less severe, and prognosis has improved primarily due to supportive care. Type III is the mildest form with onset usually of weakness in adolescence or young adulthood. SMA is an autosomal recessive disorder with deletions or mutations of the gene at the 5 q11 locus. There is no specific prevention or treatment, but current progress toward potential therapies has been substantial and several candidates including histone deacetylase (HDAC) inhibitors are under consideration for further evaluation. The authors sought to address the challenges and opportunities for testing new therapies for SMA. NEUROLOGY 2005;65:1352–1357 Spinal muscular atrophy (SMA) is a severe and often and profound hypotonia are noticeable in the first devastating neurologic disorder of infants and chil- few months of life. There is a striking discrepancy dren. The

Journal

Neurology – Wolters Kluwer Health

Published: Nov 1, 2005

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