E-LIB Suche - Ergebnisse für: Falabella, Paulo

1

RGX-381: Interim results from the first-in-human clinical t..:

Ohnsman, Christina ; Bailey, Alexander M. ; Huang, Wei...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107987 , 2024

Link: https://doi.org/10.1016/..

2

CAMPSIITE™ phase I/II/III: An interim clinical study update..:

Harmatz, Paul ; Ficicioglu, Can ; Giugliani, Roberto...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107874 , 2024

Link: https://doi.org/10.1016/..

3

OCT Biomarkers in Ocular CLN2 Disease in Patients Treated W..:

Huang, Wei Chieh ; Ohnsman, Christina M. ; Atiskova, Yevgeniya...
Investigative Ophthalmology & Visual Science. 65 (2024) 8 - p. 45 , 2024

Link: https://doi.org/10.1167/..

4

First in-human, intracisternal dosing of RGX-111, an invest..:

Wang, Raymond Y. ; Movsesyan, Nina ; Kan, Shih-hsin...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 108070 , 2024

Link: https://doi.org/10.1016/..

5

CAMPSIITE™ Phase I/II/III: An interim clinical study update..:

Pisani, Laura ; Ficicioglu, Can ; Harmatz, Paul...
Molecular Genetics and Metabolism. 141 (2024) 4 - p. 108245 , 2024

Link: https://doi.org/10.1016/..

6

Interim results from the first-in-human intracisternal dosi..:

de Souza, Carolina Fischinger Moura ; Alves, Tamires ; Duarte, Juliana...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107970 , 2024

Link: https://doi.org/10.1016/..

7

Audiology assessment of participants in CAMPSIITE™, a phase..:

Boulos, Nidal ; Roush, Patricia ; Pjetri, Ariana...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107776 , 2024

Link: https://doi.org/10.1016/..

8

RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSA..:

Gilmor, Michelle ; Wang, Raymond ; Ficicioglu, Can...
Molecular Genetics and Metabolism. 138 (2023) 3 - p. 107416 , 2023

Link: https://doi.org/10.1016/..

9

Characterization of retinal degeneration phenotype in class..:

Ohnsman, Christina ; Kim, Kwi Hye ; Huang, Wei Chieh...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107244 , 2023

Link: https://doi.org/10.1016/..

10

RGX-121 gene therapy for the treatment of neuronopathic muc..:

Harmatz, Paul ; Ficicioglu, Can H. ; Giugliani, Roberto...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107139 , 2023

Link: https://doi.org/10.1016/..

11

Daily living skills on the Vineland Adaptive Behavioral Sca..:

Phillips, Dawn ; Cho, Yoonjin ; Mulatya, Caroline...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107268 , 2023

Link: https://doi.org/10.1016/..

12

Loss of visual function associated with photoreceptor degen..:

Ohnsman, Christina ; Huang, Wei Chieh ; Atiskova, Yevgeniya...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107243 , 2023

Link: https://doi.org/10.1016/..

13

RGX-111 gene therapy for the treatment of severe mucopolysa..:

Wang, Raymond Y. ; Ficicioglu, Can H. ; Giugliani, Roberto...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107354 , 2023

Link: https://doi.org/10.1016/..

14

RGX-121 gene therapy for the treatment of severe mucopolysa..:

Harmatz, Paul ; Escolar, Maria ; Ficicioglu, Can...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S54 , 2022

Link: https://doi.org/10.1016/..

15

RGX-111 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSA..:

Wang, Raymond ; Ficicioglu, Can ; Giugliani, Roberto...
Molecular Genetics and Metabolism. 135 (2022) 4 - p. 305 , 2022

Link: https://doi.org/10.1016/..