SIETES :: 93212 citas

Resultados de la búsqueda por DISTROFIA MUSCULAR REFINAR BÚSQUEDA

1.

Dal-Ré R, Kesselheim AS, Bourgeois FT. Increasing access to FDA inspection reports on irregularities and misconduct in clinical trials. JAMA 2020:23 de abril. [Ref.ID 103599]

2.

Schuster BC, Brhlikova P, Heath J, McGettigan P. The use of validated and nonvalidated surrogate endpoints in two European Medicines Agency expedited approval pathways: A cross-sectional study of products authorised 2011–2018. PLOS Medicine 2019;16:10 de septiembre. [Ref.ID 103247]

3.

. Roche pays $1.15bn for Sarepta’s Duchenne gene therapy. PMLiVE 2019:23 de diciembre. [Ref.ID 103235]

4.

Taylor P. FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection. PMLiVE 2019:13 de diciembre. [Ref.ID 103233]

5.

Anónimo. Muscle drug determined too expensive for UK’s NHS. DIA Daily 2018:14 de agosto. [Ref.ID 102787]

6.

Anónimo. Marathon Pharmaceuticals to charge $89,000 annually for newly-approved DMD drug. DIA Daily 2017:1. [Ref.ID 101396]

7.

Anónimo. Obstacles for regulatory approval of DMD drug ataluren remain. DIA Daily 2016:2. [Ref.ID 100845]

8.

Anónimo. FDA commissioner calls for eteplirsen study to be retracted as “misleading.”. DIA Daily 2016:4. [Ref.ID 100697]

9.

Anónimo. After internal dispute, FDA approves eteplirsen for treatment of DMD. DIA Daily 2016:1. [Ref.ID 100684]

10.

Anónimo. Key FDA critic of eteplirsen leaves agency. DIA Daily 2016:2. [Ref.ID 100665]

11.

Anónimo. FDA grants experimental Huntington’s disease orphan drug status. DIA Daily 2016:1. [Ref.ID 100438]

12.

Anónimo. Patient advocates pressure FDA to approve more experimental treatments. DIA Daily 2016:2. [Ref.ID 100184]

13.

Anónimo. FDA staff critical of Sarepta’s experimental DMD drug In briefing document. DIA Daily 2016:1. [Ref.ID 100182]

14.

Anónimo. FDA declines application for DMD drug as insufficient. DIA Daily 2016:4. [Ref.ID 100013]

15.

Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset J-M, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T, for the DELOS Study Group. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet 2015;385:1748-57. [Ref.ID 99083]

16.

Anónimo. FDA provides path forward for Duchenne muscular dystrophy medicine. DIA Daily 2014:22 de abril. [Ref.ID 97450]

17.

Sherman RE, Li J, Shapley S, Robb M, Woodcock J. Expediting drug development - The FDA's new "breakthrough therapy" designation. N Engl J Med 2013;369:1877-80. [Ref.ID 96536]

18.

Hoffmann DE, Weber E. Medical marijuana and the law. N Engl J Med 2010;362:1453-7. [Ref.ID 88297]

19.

Hoffman EP. Skipping toward personalized molecular medicine. N Engl J Med 2007;357:2719-22. [Ref.ID 81935]

20.

van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, de Kimpe SJ, Ekhart PF, Venneker EH, Platenburg GJ, Verschuuren JJ, van Ommen GJB. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007;357:2677-86. [Ref.ID 81934]