期刊
JOURNAL OF CHILD NEUROLOGY
卷 22, 期 8, 页码 957-966出版社
SAGE PUBLICATIONS INC
DOI: 10.1177/0883073807305665
关键词
spinal muscular atrophy; clinical trials; historie deacetylase inhibitors
资金
- NICHD NIH HHS [R01 HD054599, R01 HD054599-05] Funding Source: Medline
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) subtyes 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.
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