Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases RESEARCH 3-hydroxy-3-methylglutaryl-coenzyme A lyase deficiency: one disease - many faces Sarah C. Grünert and Jörn Oliver Sass Orphanet Journal of Rare Diseases 2020, 15:48 | Published on: 14 February 2020 Full Text | PDF REVIEW The effect of human gene therapy for RPE65-associated Leber’s congenital amaurosis on visual function: a systematic review and meta-analysis Xue Wang, Chaofeng Yu, Radouil T. Tzekov, Yihua Zhu and Wensheng Li Orphanet Journal of Rare Diseases 2020, 15:49 | Published on: 14 February 2020 Full Text | PDF

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