PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
2 of 2 results sorted by: Most RecentSee all results in PubMed (2)
Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent.
Mol Ther. 2016. PMID: 26860981 Free PMC article.
Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1.
J Cell Mol Med. 2015. PMID: 26095024 Free PMC article. Review.