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项与 Metabolism(阿尔法分子科技) 相关的临床试验Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2 years.
The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population, either routinely or for future clinical trials. The investigators also aim to collect the total costs associated with ASI in order to propose a first prospective medico-economic study in France.
100 项与 Metabolism(阿尔法分子科技) 相关的临床结果
100 项与 Metabolism(阿尔法分子科技) 相关的转化医学
100 项与 Metabolism(阿尔法分子科技) 相关的专利(医药)
100 项与 Metabolism(阿尔法分子科技) 相关的药物交易