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4. Characterising a homozygous two‐exon deletion in UQCRH: comparing human and mouse phenotypes. Issue 12 (8th November 2021)

5. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease. Issue 3 (28th March 2013)

7. Genotype–phenotype specificity in Menke–Hennekam syndrome caused by missense variants in exon 30 or 31 of CREBBP. Issue 6 (20th March 2019)

8. High dose genistein in Sanfilippo syndrome: A randomised controlled trial. Issue 5 (13th June 2021)

9. Irbesartan in Marfan syndrome (AIMS): a double-blind, placebo-controlled randomised trial. Issue 10216 (21st December 2019)