TY - JOUR
T1 - Perspectives on clinical trials in spinal muscular atrophy
AU - Swoboda, Kathryn J.
AU - Kissel, John T.
AU - Crawford, Thomas O.
AU - Bromberg, Mark B.
AU - Acsadi, Gyula
AU - D'Anjou, Guy
AU - Krosschell, Kristin J.
AU - Reyna, Sandra P.
AU - Schroth, Mary K.
AU - Scott, Charles B.
AU - Simard, Louise R.
PY - 2007/8
Y1 - 2007/8
N2 - Spinal muscular atrophy is one of the most heterogeneous of the single-gene neuromuscular disorders. The broad spectrum of severity, with onset from the prenatal period to adulthood, presents unique challenges in the design and implementation of clinical trials. The clinical classification of subjects into severe (type 1), intermediate (type 2), and mild (type 3) subtypes has proved useful both in enhancing communication among clinicians internationally and in forging the collaborative development of outcome measures for clinical trials. Ideally, clinical trial design in spinal muscular atrophy must take into account the spinal muscular atrophy type, patient age, severity-of-affection status, nature of the therapeutic approach, timing of the proposed intervention relative to disease progression, and relative homogeneity of the cohort to be studied. Following is an overview of the challenges and opportunities, current and future therapeutic strategies, and progress to date in clinical trials in spinal muscular atrophy.
AB - Spinal muscular atrophy is one of the most heterogeneous of the single-gene neuromuscular disorders. The broad spectrum of severity, with onset from the prenatal period to adulthood, presents unique challenges in the design and implementation of clinical trials. The clinical classification of subjects into severe (type 1), intermediate (type 2), and mild (type 3) subtypes has proved useful both in enhancing communication among clinicians internationally and in forging the collaborative development of outcome measures for clinical trials. Ideally, clinical trial design in spinal muscular atrophy must take into account the spinal muscular atrophy type, patient age, severity-of-affection status, nature of the therapeutic approach, timing of the proposed intervention relative to disease progression, and relative homogeneity of the cohort to be studied. Following is an overview of the challenges and opportunities, current and future therapeutic strategies, and progress to date in clinical trials in spinal muscular atrophy.
KW - Clinical trials
KW - Histone deacetylase inhibitors
KW - Spinal muscular atrophy
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U2 - 10.1177/0883073807305665
DO - 10.1177/0883073807305665
M3 - Review article
C2 - 17761650
AN - SCOPUS:34548154298
SN - 0883-0738
VL - 22
SP - 957
EP - 966
JO - Journal of child neurology
JF - Journal of child neurology
IS - 8
ER -