Suchergebnisse - "Gidaro, Teresa"

PubPharm (93)

1

Clinical and genetic features of patients suffering from CMT4J

enthalten in: Journal of neurology | 2024

von Beloribi-Djefaflia, S. | Morales, R. | Fatehi, F. | +10

2

The Clinical Development of Taldefgrobep Alfa : An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy

enthalten in: Neurology and therapy | 2024

von Muntoni, F. | Byrne, B. | McMillan, H. | +114

3

Pathogenic DPAGT1 variants in limb‐girdle congenital myasthenic syndrome (LG‐CMS) associated with tubular aggregates and ORAI1 hypoglycosylation

enthalten in: Neuropathology and Applied Neurobiology | 2024

von Brande, L. | Bauché, S. | Pérez‐Guàrdia, L. | +13

4

Pathogenic DPAGT1 variants in limb-girdle congenital myasthenic syndrome (LG-CMS) associated with tubular aggregates and ORAI1 hypoglycosylation

enthalten in: Neuropathology and applied neurobiology | 2023

von Brande, L. | Bauché, S. | Pérez-Guàrdia, L. | +13

5

Clinical and genetic features of patients suffering from CMT4J

enthalten in: Journal of neurology | 2023

von Beloribi-Djefaflia, S. | Morales, R. | Fatehi, F. | +10

6

Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

enthalten in: Neuromuscular disorders : NMD | 2023

von Mercuri, E. | Seferian, A. | Servais, L. | +32

7

A negative feedback loop between fibroadipogenic progenitors and muscle fibres involving endothelin promotes human muscle fibrosis

enthalten in: Journal of cachexia, sarcopenia and muscle | 2022

von Bensalah, M. | Muraine, L. | Boulinguiez, A. | +15

8

Convergence of patient- and physician-reported outcomes in the French National Registry of Facioscapulohumeral Dystrophy

enthalten in: Orphanet journal of rare diseases | 2022

von Sanson, B. | Stalens, C. | Guien, C. | +114

9

Home-based gait analysis as an exploratory endpoint during a multicenter phase 1 trial in limb girdle muscular dystrophy type R2 and facioscapulohumeral muscular dystrophy

enthalten in: Muscle & nerve | 2022

von Gidaro, T. | Gasnier, E. | Annoussamy, M. | +9

10

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2) : a phase 3, double-blind, randomised, placebo-controlled trial

enthalten in: The Lancet. Neurology | 2022

von Mercuri, E. | Deconinck, N. | Mazzone, E. | +121

CommentOn: Lancet Neurol. 2022 Jan;21(1):23-24. - PMID 34942130

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