Clinical studies in lysosomal storage diseases: Past, present, and future (Q27025186)

language of work or name

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2013

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Rare diseases (Austin, Tex.)

published in

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volume

1

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page(s)

e26690

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The ethics of placebo-controlled trials--a middle ground

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Therapeutic approaches for lysosomal storage diseases

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Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

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A randomized study of alglucosidase alfa in late-onset Pompe's disease

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28 September 2017

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Clinical research for rare disease: opportunities, challenges, and solutions

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21 January 2018

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Reforming accelerated approval

28 November 2018

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12 December 2020

Europe sets out to reform its clinical trial rules

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12 December 2020

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

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12 December 2020

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

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Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

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12 December 2020