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5. The GENESIS database and tools: A decade of discovery in Mendelian genomics

12. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

20. Neuronal intranuclear inclusion disease in New Zealand: A novel discovery

24. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

26. Sulfated disaccharide protects membrane and DNA damages from arginine-rich dipeptide repeats in ALS

32. Biallelic COQ4 Variants in Hereditary Spastic Paraplegia: Clinical and Molecular Characterization

34. Renal function is associated with 1-month and 1-year mortality in patients with ischemic stroke

35. Phenotypic Heterogeneity in Patients with Mutations in the Mitochondrial Complex I Assembly Gene NDUFAF5

39. Cholesterol Levels Are Associated with 30-day Mortality from Ischemic Stroke in Dialysis Patients

41. Biallelic COQ4 Variants in Hereditary Spastic Paraplegia: Clinical and Molecular Characterization.

44. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

50. Monogenic Causes in Familial Stroke Across Intracerebral Hemorrhage and Ischemic Stroke Subtypes Identified by Whole-Exome Sequencing

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