Results 171 to 180 of about 640,820 (294)

Evaluating Innovative Health Programs: Lessons for Health Policy [PDF]

open access: yes
The Global Development Network’s (GDN) project “Evaluating Innovative Health Programs” (EIHP), funded by the Bill & Melinda Gates Foundation, seeks to inform policy on the effectiveness of health solutions that have the potential to improve health ...
Thomas, Ranjeeta   +2 more
core  

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

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

Global Stories: Child Health and Mortality Prevention Surveillance Network (CHAMPS) Program

open access: yes
Every year, six million children die before their 5th birthday. CDC and partners are working to answer the critical question of why. CDC\u2019s Division of High-Consequence Pathogens and Pathology (DHCPP) out of the National Center for Emerging and ...

core  

The Role of Childhood Health for the Intergenerational Transmission of Human Capital: Evidence from Administrative Data [PDF]

open access: yes
We use unique administrative German data to examine the role of childhood health for the intergenerational transmission of human capital. Specifically, we examine the extent to which a comprehensive list of health conditions – diagnosed by government ...
Schunk, Daniel, Salm, Martin
core  

Sertraline Treatment Can Mimic Niemann‐Pick Type C Biomarker Profile: A Diagnostic Pitfall

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Oxysterols (cholestane‐3β,5α,6β‐triol and 7‐ketocholesterol) and N‐palmitoyl‐O‐phosphocholineserine (PPCS) are sensitive biomarkers for Niemann‐Pick disease type C (NPC) screening. However, false‐positive results occur, with a biomarker profile suggestive of NPC despite the absence of pathogenic variants in genes involved in NPC or ...
Maria Makrygianni   +19 more
wiley   +1 more source

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