69 results on '"Peña, Loren"'
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52. NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients
53. Late onset Leigh syndrome mimicking central nervous system vasculitis
54. Episodic myoglobinuria in a primary gamma-sarcoglycanopathy
55. De Novo Truncating Variants in ASXL2 Are Associated with a Unique and Recognizable Clinical Phenotype
56. Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients
57. Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients
58. Clinical characteristics and genotypes in the ADVANCE baseline dataset, a comprehensive cohort of us children and youth with Pompe disease
59. 52-week efficacy and safety profile of alglucosidase alfa produced at 4000 liter scale in US patients with Pompe disease: ADVANCE, a phase 4 open-label prospective study
60. Neurologic considerations in propionic acidemia
61. Chronic management and health supervision of individuals with propionic acidemia
62. Premature Pubarche in Children with Pompe Disease
63. A phase 4 prospective study in patients with adult Pompe disease treated with alglucosidase alfa
64. Differentiation-Dependent Chromatin Rearrangement Coincides with Activation of Human Papillomavirus Type 31 Late Gene Expression
65. NEO1 and NEO-EXT studies: Long-term safety of repeat avalglucosidase alfa dosing for 4.5 years in late-onset Pompe disease patients
66. Follow-up of patients with short-chain acyl-CoA dehydrogenase and isobutyryl-CoA dehydrogenase deficiencies identified through newborn screening: one center’s experience
67. Acute management of propionic acidemia
68. Natural history of propionic acidemia
69. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1
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