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Cancers|October 14, 2022
Differences and Similarities in the Pattern of Early Metabolic and Morphologic Response after Induction Chemo-Immunotherapy versus Induction Chemotherapy Alone in Locally Advanced Squamous Cell Head and Neck CancerMichael Beck, Sabine Semrau, Marlen Haderlein, et al.
Pharmacoepidemiology and Drug Safety|December 16, 2011
Source document verification in the Mucopolysaccharidosis Type I RegistryKarien Verhulst, Laura Artiles-Carloni, Michael Beck, et al.
The Journal of Pediatrics|May 2, 2006
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension studyPaul Harmatz, Roberto Giugliani, Ida Schwartz, et al.
Human Mutation|April 27, 2007
Mutational analysis of 105 mucopolysaccharidosis type VI patientsLitsa Karageorgos, Doug A Brooks, Anthony Pollard, et al.
Journal of Inherited Metabolic Disease|May 31, 2018
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trialLine Borgwardt, Nathalie Guffon, Yasmina Amraoui, et al.
Annals of Hematology|May 4, 2021
Clinical outcomes in patients with Philadelphia chromosome-positive leukemia treated with ponatinib in routine clinical practice-data from a Belgian registryTimothy Devos, Violaine Havelange, Koen Theunissen, et al.
American Journal of Human Genetics|October 10, 2007
Mutations in TOPORS cause autosomal dominant retinitis pigmentosa with perivascular retinal pigment epithelium atrophyChristina F Chakarova, Myrto G Papaioannou, Hemant Khanna, et al.
Pediatric Research|June 23, 2009
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseasePriya S Kishnani, Deya Corzo, Nancy D Leslie, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|December 15, 2010
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndromeJoseph Muenzer, Michael Beck, Christine M Eng, et al.
Molecular Genetics and Metabolism|May 27, 2008
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatasePaul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, et al.
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