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84 results on '"Pestronk, A"'

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1. Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern

2. Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy

3. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease

4. Ophthalmoparesis as an unusual manifestation of anti-3‑hydroxy-3-methyl-glutaryl-coenzyme A reductase antibody-associated myopathies.

9. Clinical and genetic characterization of manifesting carriers of DMD mutations

22. Sarcoidosis, granulomas and myopathy syndromes: A clinical-pathology review.

29. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.

31. T.P.5.04 Randomized, double-blind, controlled study to compare efficacy and tolerability of standard daily prednisone regime with a novel intermittent high dose regime in ambulant boys with Duchenne muscular dystrophy

34. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.

35. Homozygous recessive MYH2 mutation mimicking dominant MYH2 associated myopathy.

36. 93P The International Clinical Outcome Study for Dysferlinopathy II: validation of motor outcome measures in a new patient cohort.

37. 81P Minimal clinically important differences in dysferlinopathy from the 10-year, multicenter Jain Clinical Outcome Study.

40. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

45. Paraneoplastic myopathy: response to intravenous immunoglobulin

46. Clinical and genetic heterogeneity in chromosome 9p associated hereditary inclusion body myopathy: exclusion of GNE and three other candidate genes

47. Complement 3 deficiency and oral prednisolone improve strength and prolong survival of laminin α2-deficient mice

48. An unusual pathologic feature associated with dermatomyositis

49. LIMB-GIRDLE MUSCULAR DYSTROPHY I: P.13Rasch analysis of the individualised neuromuscular Quality of Life Questionnaire administered to patients with dysferlinopathy.

50. LIMB-GIRDLE MUSCULAR DYSTROPHY I: P.12Clinical outcome study in dysferlinopathy: medical comorbidities and polytherapy in a large population of dysferlinopathy patients.

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