Suchergebnisse - "Bratkovic, Drago"

PubPharm (54)

1

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

2

Open-label, single-center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I

enthalten in: Journal of inherited metabolic disease | 2024

von Bratkovic, D. | Gravance, C. | Ketteridge, D. | +5

3

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

4

Fabry-specific treatment in Australia : time to align eligibility criteria with international best practices

enthalten in: Internal medicine journal | 2024

von Nicholls, K. | Denaro, C. | Tchan, M. | +5

5

Liver transplantation for acute-on-chronic liver failure due to carnitine palmitoyl transferase (CPT) 1A deficiency

enthalten in: Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society | 2024

von Smart, C. | Bratkovic, D. | Muller, K.

6

Open‐label, single‐center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I

enthalten in: Journal of Inherited Metabolic Disease | 2024

von Bratkovic, D. | Gravance, C. | Ketteridge, D. | +5

7

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 | 2023

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

8

The Australian Genomics Mitochondrial Flagship: A National Program Delivering Mitochondrial Diagnoses

enthalten in: bioRxiv.org | 2023

von Rius, R. | Compton, A. | Baker, N. | +64

9

Corrigendum to "Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II"

enthalten in: Molecular genetics and metabolism | 2023

von Muenzer, J. | Burton, B. | Harmatz, P. | +12

ErratumFor: Mol Genet Metab. 2022 Sep-Oct;137(1-2):92-103. - PMID 35961250

10

Corrigendum to : Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

enthalten in: Molecular genetics and metabolism | 2023

von Muenzer, J. | Burton, B. | Harmatz, P. | +12

ErratumFor: Mol Genet Metab. 2022 Sep-Oct;137(1-2):127-139. - PMID 36027721

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