Harnessing the power of the patient perspective for rare disease therapeutics
- PMID: 30143562
- DOI: 10.1212/WNL.0000000000006230
Harnessing the power of the patient perspective for rare disease therapeutics
Comment on
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Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA).Neurology. 2018 Sep 25;91(13):e1206-e1214. doi: 10.1212/WNL.0000000000006241. Epub 2018 Aug 24. Neurology. 2018. PMID: 30143566 Free PMC article.
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