Results 121 to 130 of about 1,722 (231)
Nusinersen versus sham control in later-onset spinal muscular atrophy [PDF]
Connolly, A. M., et al,
core +1 more source
ABSTRACT Introduction Children with medical complexity are at risk of pulmonary injury due to chronic pulmonary aspiration. Our study aimed to determine the effect of multidisciplinary evaluation and periodic follow‐up of aerodigestive disorders on the final status of the respiratory conditions.
Füsun Ünal +9 more
wiley +1 more source
Hirayama disease: an uncommon cause of motor neuron disease. [PDF]
Gonçalves TAP +4 more
europepmc +1 more source
ABSTRACT The objective was to prepare guidelines to perform the current optimum treatment by organizing effective and efficient treatments of hemangiomas and vascular malformations, confirming the safety, and systematizing treatment, employing evidence‐based medicine techniques and aimed at improvement of the outcomes.
Yoshiaki Kinoshita +116 more
wiley +1 more source
DYNC1H1 in Spinal Muscular Atrophy: Diagnostic Findings From Two Families and a Comprehensive Review of Its Role in Neuromuscular and Neurodevelopmental Disorders. [PDF]
Namdari M +10 more
europepmc +1 more source
ABSTRACT Background School‐aged children with neurodisability and oropharyngeal dysphagia who need mealtime assistance have an increased risk of premature death. Speech & Language Therapists (SLTs) provide assessment and recommendations to optimise mealtime nutrition and hydration, but little is known about current clinical practice including mealtime ...
Sally Morgan +3 more
wiley +1 more source
Effects of physical therapy on motor ability in patients with spinal muscular atrophy III: a study protocol for a randomised controlled trial. [PDF]
Zhang J, Wang B, He J, Zhu L, Dong X.
europepmc +1 more source
Enteric Nervous System Damage by Food Contaminants: A Pathway to Neurodegeneration?
ABSTRACT The enteric nervous system (ENS), a key component of the gut–brain axis, has emerged as a critical player in the pathogenesis of Parkinson's disease (PD). It is the first neural system exposed to food contaminants (FCs)—a diverse group of ubiquitous toxic compounds fortuitously present in food derived from production, processing, storage, or ...
Helena Ramos +3 more
wiley +1 more source
ABSTRACT A frequently cited concern regarding patient‐as‐own‐control trial designs in rare disease is the potential for placebo and related effects to inflate apparent treatment efficacy. Whether this concern is disqualifying or manageable has not been systematically evaluated.
Marshall L. Summar, Janet Woodcock
wiley +1 more source
Body Weight and Range of Motion as Predictors of Trunk Asymmetry in Children With Spinal Muscular Atrophy: A Prospective Functional Assessment. [PDF]
Gajewska E, Bieniaszewska A, Sobieska M.
europepmc +1 more source

