12 results on '"McBride, Kim L."'
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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. 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)
5. 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)
6. Evaluation of biomarkers for Sanfilippo syndrome
7. Natural history of echocardiographic abnormalities in mucopolysaccharidosis III
8. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect
9. Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB
10. Prospective natural history study of mucopolysaccharidosis types IIIA and IIIB (Sanfilippo syndrome)
11. Cardiac teratogenicity in mouse maternal phenylketonuria: Defining phenotype parameters and genetic background influences
12. Heritability of plasma amino acid levels in different nutritional states
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