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52. Evaluation of Postural Control and Falls in Individuals with Myotonic Dystrophy Type 1

53. Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience.

55. Transcriptome alterations in myotonic dystrophy skeletal muscle and heart

58. Facioscapulohumeral muscular dystrophy functional composite outcome measure

59. Recruiting for an International Rare Disease Clinical Trial Readiness Study during the COVID‐19 pandemic: Challenges and solutions.

60. Dose-Dependent Regulation of Alternative Splicing by MBNL Proteins Reveals Biomarkers for Myotonic Dystrophy

63. The Myotonic Dystrophy Health Index: Correlations with Clinical Tests and Patient Function

64. Prospective Study of Muscle Cramps in Charcot Marie Tooth Disease

71. Study Design of a Phase 1/2a Trial with ISIS-DMPKRx for the Treatment of Myotonic Dystrophy Type 1 (P3.167)

73. Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS.

74. Myotonic dystrophy health index: Correlations with clinical tests and patient function

75. An Open-Label Trial of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-1/rhIGFBP-3) in Myotonic Dystrophy Type 1

79. Contributors

82. Contributors

84. SupplementalFile1_Table_1 – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

85. SupplementaryFile3_Managed_Frequencies – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

86. SupplementaryFile2_Needs_Frequencies – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

87. SupplementaryFile3_Managed_Frequencies – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

88. Additional file 1: of Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study

89. SupplementalFile1_Table_1 – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

90. Additional file 1: of Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study

91. SupplementaryFile2_Needs_Frequencies – Supplemental material for Needs management in families affected by childhood-onset dystrophinopathies

93. Contributors

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