FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis

Author:

Garbade Sven F.,Zielonka Matthias,Mechler KonstantinORCID,Kölker Stefan,Hoffmann Georg F.,Staufner Christian,Mengel Eugen,Ries MarkusORCID

Publisher

Public Library of Science (PLoS)

Subject

Multidisciplinary

Reference49 articles.

1. Pressure for drug development in lysosomal storage disorders—a quantitative analysis thirty years beyond the US orphan drug act;K Mechler;Orphanet journal of rare diseases,2015

2. Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis;M Zielonka;Genetics in medicine: official journal of the American College of Medical Genetics,2017

3. A cross-sectional quantitative analysis of the natural history of Farber disease: an ultra-orphan condition with rheumatologic and neurological cardinal disease features;M Zielonka;Genetics in medicine: official journal of the American College of Medical Genetics,2018

4. A cross-sectional quantitative analysis of the natural history of free sialic acid storage disease-an ultra-orphan multisystemic lysosomal storage disorder;M Zielonka;Genetics in medicine: official journal of the American College of Medical Genetics,2018

5. Quantitative natural history characterization in a cohort of 142 published cases of patients with galactosialidosis-A cross-sectional study;T Sláma;Journal of inherited metabolic disease,2019

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