Items where Author is "Straub, V"

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Number of items: 9.

Article

Willmann, R and Lee, J and Turner, C and Nagaraju, K and Aartsma-Rus, A and Wells, D J and Wagner, K R and Csimma, C and Straub, V and Grounds, M D and De Luca, A (2020) Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT). Disease Models and Mechanisms, 13 (2).

Wagner, K R and De Luca, A and Caizergues, D and Dowling, J and Goemans, N and Gordish-Dressman, H and Grounds, M D and Kelly, M and Mayhew, A and McNally, E M and Zoetis, T and Lee, J and Turner, C and Wells, D J and Csimma, C and Straub, V (2019) A decade of optimizing drug development for rare neuromuscular disorders through TACT. Nature Reviews Drug Discovery, 19 (1-2).

Heslop, E and Csimma, C and Straub, V and McCall, J and Nagaraju, K and Wagner, K R and Caizergues, D and Korinthenberg, R and Flanigan, K M and Kaufmann, P and McNeil, E and Mendell, J and Hesterlee, S and Wells, D J (2015) The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. ORPHANET JOURNAL OF RARE DISEASES, 10 (49).

Lynn, S and Aartsma-Rus, A and Furlong, P and Goemans, N and De Luca, A and Mayhew, A and McDonald, C and Mercuri, E and Muntoni, F and Pohlschmidt, M and Verschuuren, J and Voit, T and Vroom, E and Wells, D J and Straub, V (2015) Measuring Clinical Effectiveness of Medicinal Products for the Treatment of Duchenne Muscular Dystrophy. Neuromuscular Disorders, 25 (1). pp. 96-105.

Wells, D J and Heslop, E and Csimma, C and Straub, V and McCall, J and Nagaraju, K and Wagner, K and Caizergues, D and Korinthenberg, R and Flanigan, K and Mendell, J and Kelly, M and Kaufmann, P and McNeil, E and Robertson, A and Johnston, L and Bushby, K (2015) THE TREAT-NMD ADVISORY COMMITTEE FOR THERAPEUTICS (TACT): AN INNOVATIVE DE-RISKING MODEL TO FOSTER ORPHAN DRUG DEVELOPMENT. Neuromuscular Disorders, 25. S271-S271.

Cirak, S and Arechavala-Gomeza, V and Guglieri, M and Feng, L and Torelli, S and Anthony, K and Abbs, S and Garralda, M E and Wells, DJ and Dickson, G and Wood, M J A and Wilton, S D and Straub, V and Shrewsbury, S and Sewry, C and Morgan, J E and Bushby, K and Muntoni, F (2011) Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study. Lancet, 378 (9791). pp. 595-605.

Amthor, H and Egelhof, T and McKinnell, I and Ladd, M E and Janssen, I and Weber, J and Sinn, H and Schrenk, H H and Forsting, M and Voit, T and Straub, V (2004) Albumin targeting of damaged muscle fibres in the mdx mouse can be monitored by MRI. Neuromuscular Disorders, 14 (12). pp. 791-796.

Conference or Workshop Item

Heslop, E and Bushby, K and Csimma, C and Straub, V and Wells, D J (2012) MOVING FORWARD WITH TACT: 2 YEARS ON. In: UNSPECIFIED.

Cirak, S and Arechavala-Gomeza, V and Guglieri, M and Feng, L and Torelli, S and Anthony, K and Garralda, M E and Wells, D J and Dickson, G and Wood, M J A and Wilton, S D and Straub, V and Shrewsbury, S B and Sewry, C and Morgan, J E and Bushby, K and Muntoni, F (2011) EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT. In: UNSPECIFIED.

This list was generated on Sat Sep 25 07:28:34 2021 BST.