BACKGROUND: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder and a leading genetic cause of infant death worldwide. However, there is no routine screening program for SMA in the UK. Lack of treatments and the inability of…
Introduction: Awareness for paediatric palliative care has resulted in the impetus for paediatrician-led palliative care services across Malaysia. However, there is paucity of local data on patients receiving hospital-based paediatric palliative…
BACKGROUND: Discomfort exists discussing goals of care (GOC) with families of children with advanced life-threatening illnesses. There also exists important variability in the management of these patients. OBJECTIVE(S): This study seeks to explore…
Objective: Our aim was to describe the clinical evolution and needs of children with spinal muscular atrophy type I treated in a domiciliary palliative care program. Method: We undertook a retrospective chart review of nine consecutive patients.…
AIM: Spinal muscular atrophy (SMA) type 1 is a relatively common, untreatable and invariably fatal neuromuscular disorder of early childhood. Psychosocial care is vital in management of families affected by this disease. There are few studies…
BACKGROUND AND AIMS:: Children with severe spinal muscular atrophy have complex care needs due to progressive muscle weakness, eventually leading to respiratory failure. To design a care system adapted to families' needs, more knowledge about…
Aims A proportion of children with neurodisability will have life-limiting conditions, whereby their complex continuing needs increase the intricacy and urgency for providing comprehensive supportive care to these families. Equal access to universal…
The aim of this prospective longitudinal multi centric study was to evaluate the correlation between the Hammersmith Functional Motor Scale and the 20 item version of the Motor Function Measure in non ambulant SMA children and adults at baseline and…
STUDY PURPOSE: The purpose of this qualitative study was to understand, from the parent perspective, the experience of the family whose child has Type 1 spinal muscular atrophy (Type 1 SMA), in the emergency center, hospital, and clinical care…
BACKGROUND: Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory…
Purpose: Children with severe neurological disabilities are at an increased risk of acute, life-threatening events. We assessed physicians' attitudes when making decisions in these situations.
