15 results on '"Truxal, Kristen"'
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2. Interim results of Transpher A, a multicentre, single-dose, phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
3. Longitudinal MRI brain volume changes over one year in children with mucopolysaccharidosis types IIIA and IIIB
4. Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)
5. Evaluating the mutational spectrum of SIN3A alterations: a case series of patients profiled by next generation sequencing
6. Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
7. Robust diagnostic yield and candidate gene discovery through paired exome analysis in vascular malformation and overgrowth
8. Outcomes of in-house rapid genome sequencing at a Children’s Hospital
9. Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)
10. Safety, tolerability and preliminary evidence of biopotency in Transpher B, a multicenter, single-dose, phase 1/2 clinical trial of ABO-101 gene therapy for Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB)
11. Evaluation of biomarkers for Sanfilippo syndrome
12. Natural history of echocardiographic abnormalities in mucopolysaccharidosis III
13. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect
14. Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB
15. Prospective natural history study of mucopolysaccharidosis types IIIA and IIIB (Sanfilippo syndrome)
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