Results 191 to 200 of about 473,853 (319)

Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane   +19 more
wiley   +1 more source

Fiscal Year 2005 Performance and Accountability Report

open access: yes, 2005
Other written product issued by the Government Accountability Office with an abstract that begins "Presented is GAO's performance and accountability report for fiscal year 2005.
United States. Government Accountability Office.
core  

Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN‐Digenic Myopathy

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SRPK3/TTN‐digenic myopathy was recently established as a skeletal muscle myopathy caused by digenic inheritance. This study characterizes the early clinical presentation of SRPK3/TTN‐digenic myopathy in one previously reported and seven newly identified pediatric patients.
Rotem Orbach   +23 more
wiley   +1 more source

District of Columbia: FY 2003 Performance Report Shows Continued Improvements

open access: yes, 2004
Correspondence issued by the Government Accountability Office with an abstract that begins "This is the fifth consecutive year that we have reviewed the District of Columbia's performance accountability report as mandated by the Federal Payment ...
United States. Government Accountability Office.
core  

Compound Heterozygote Friedreich Ataxia Patients With Covert Proximal FXN Gene Deletions

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT We present Friedreich ataxia patients with frataxin gene deletions. Data and records were collected at the Children's Hospital of Philadelphia from patients enrolled in the FACOMS natural history study. Patients with proximal deletions initially diagnosed with only one GAA expanded allele had more severe disease than their homozygous expansion
Michael P. Lazaropoulos   +5 more
wiley   +1 more source

Performance Measurement for Surgery at National Level in China. [PDF]

open access: yesHealth Care Sci
Zhang T   +9 more
europepmc   +1 more source

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