Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design.

نویسندگان

  • P Kaufmann
  • F Muntoni
چکیده

Advances in preclinical SMA research have identified several candidate treatments. Early clinical trials evaluating these medications are underway or in the planning stage. At the NINDS Workshop on SMA in September 2004 in Washington DC, discussions started within the international SMA community to identify features that should be common to SMA clinical trials [1]. Having a set of design features and outcome measures common to clinical trials would allow for

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عنوان ژورنال:
  • Neuromuscular disorders : NMD

دوره 17 6  شماره 

صفحات  -

تاریخ انتشار 2007