13 results on '"Paulo Falabella"'
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2. Loss of visual function associated with photoreceptor degeneration in CLN2 disease
3. Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an retinal model
4. RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in human study
5. RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study
6. Daily living skills on the Vineland Adaptive Behavioral Scale version 2 (VABS-II) in neuronopathic mucopolysaccharidosis type II (MPS II)
7. RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE I (MPS I): INTERIM ANALYSIS OF DATA FROM A PHASE 1/2 STUDY
8. RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial
9. First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up
10. RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestations
11. Intracisternal administration of AAV9 gene therapies to target the central nervous system
12. RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study
13. RGX-121 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE II (MPS II): INTERIM ANALYSIS OF DATA FROM A PHASE 1/2 STUDY
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