Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

Autor: Treadwell, Marsha, Harmatz, Paul R., Burton, Barbara K., Mitchell, John J., Muschol, Nicole, Jones, Simon A., Pastores, Gregory M., Lau, Heather A., Sparkes, Rebecca, Sutton, V. Reid, Meesen, Bianca, Haller, Christine A., Shaywitz, Adam J., Gold, Jeffrey I.

Publikováno v: Journal of Inborn Errors of Metabolism and Screening v.5 2017
Journal of Inborn Errors of Metabolism and Screening
Instituto Genética para Todos (IGPT)
instacron:IGPT
Journal of Inborn Errors of Metabolism and Screening, Volume: 5, Article number: e170008, Published: 16 MAY 2019

Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-report

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::34faf9e4cb3810c3e038b5fe439848a2
http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100803

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Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses

Autor: Scarpa, Maurizio, Harmatz, Paul R., Meesen, Bianca, Giugliani, Roberto

Publikováno v: Journal of Inborn Errors of Metabolism and Screening; December 2018, Vol. 6 Issue: 1

The regulator's perspective: How should new therapies and follow-on products for MS be clinically evaluated in the future?

Autor: Crommelin DJ; Department of Pharmaceutics, Pharmaceutical Sciences, Utrecht Institute for Pharmaceutical Sciences (UIPS), Faculty of Sciences, Utrecht University, Utrecht, The Netherlands D.J.A.Crommelin@uu.nl., Broich K; President and Head of the Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM), Bonn, Germany., Holloway C; European Regulatory Consultant, Chief Scientific Officer of ERA Consulting GmbH, Walsrode, Germany., Meesen B; Managing Director at Ismar Healthcare, Lier, Belgium., Lizrova Preiningerova J; Department of Neurology and Center of Clinical Neuroscience, First Faculty of Medicine, Charles University, Prague, Czech Republic., Prugnaud JL; Expert Involved in the Development of Recommendations Related to Drug Registrations, Paris, France., Silva-Lima B; iMed.ULisboa, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.

Publikováno v: Multiple sclerosis (Houndmills, Basingstoke, England) [Mult Scler] 2016 Aug; Vol. 22 (2 Suppl), pp. 47-59.