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3. A splice-switching oligonucleotide treatment ameliorates glycogen storage disease type 1a in mice with G6PC c.648G>T

8. Clinical Utility of Synthesized 18-Lead Electrocardiography.

10. Clinical features of autosomal recessive polycystic kidney disease in the Japanese population and analysis of splicing in PKHD1 gene for determination of phenotypes

12. Systematic Review of Genotype-Phenotype Correlations in Frasier Syndrome

14. Differential metabolic secretion between muscular dystrophy mouse-derived spindle cell sarcomas and rhabdomyosarcomas drives tumor type development.

15. Common risk variants in NPHS1 and TNFSF15 are associated with childhood steroid-sensitive nephrotic syndrome

22. Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy.

25. A splice-switching oligonucleotide treatment ameliorates glycogen storage disease type 1a with G6PC c.648G>T

36. Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5

39. A case of infantile Barth syndrome with severe heart failure: Importance of splicing variants in the TAZ gene

41. A case of infantile Barth syndrome with severe heart failure: Importance of splicing variants in the TAZ gene

48. Natural history of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy: a retrospective study at a single referral center in Japan

49. Intragenic mutations in SMN1 may contribute more significantly to clinical severity than SMN2 copy numbers in some spinal muscular atrophy (SMA) patients

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