Suchergebnisse - "Kishnani, Priya S"

PubPharm (538)

1

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease : a phase III open-label extension study (ATB200-07)

enthalten in: Journal of neurology | 2024

von Schoser, B. | Kishnani, P. | Bratkovic, D. | +81

2

Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy

enthalten in: Europäisches Patentamt | 2024

von KISHNANI PRIYA S | KAZI ZOHEB B | DESAI ANKIT K

3

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease : an open-label phase I/II study (ATB200-02)

enthalten in: Journal of neurology | 2024

von Byrne, B. | Schoser, B. | Kishnani, P. | +17

4

Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia : results from a global registry

enthalten in: Orphanet journal of rare diseases | 2024

von Kishnani, P. | Martos-Moreno, G. | Linglart, A. | +8

5

Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry

enthalten in: Orphanet journal of rare diseases | 2024

von Kishnani, P. | Martos-Moreno, G. | Linglart, A. | +8

6

Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa : Meaningful change analyses from the Phase 3 COMET trial

enthalten in: Molecular genetics and metabolism | 2024

von Toscano, A. | Pollissard, L. | Msihid, J. | +10

7

Severe CNS involvement in a subset of long-term treated children with infantile-onset Pompe disease

enthalten in: Molecular genetics and metabolism | 2024

von Kenney-Jung, D. | Korlimarla, A. | Spiridigliozzi, G. | +11

8

Underrepresentation of Diverse Ancestries Drives Uncertainty in Genetic Variants Found in Cardiomyopathy-Associated Genes

enthalten in: JACC. Advances | 2024

von Rosamilia, M. | Markunas, A. | Kishnani, P. | +1

9

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

enthalten in: Journal of patient-reported outcomes | 2024

von Kishnani, P. | Shohet, S. | Raza, S. | +7

10

Gene therapy for glycogen storage diseases

enthalten in: Journal of inherited metabolic disease | 2024

von Koeberl, D. | Koch, R. | Lim, J. | +4

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