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Spinal muscular atrophy

Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy

Objective: To quantify the economic and health-related quality of life (HRQoL) burden incurred by households with a child affected by spinal muscular atrophy (SMA). Methods: Hospital records, insurance claims, and detailed resource use questionnaires completed by caregivers were used to capture the direct and indirect costs to households of 40 children affected by SMA I, II, and III in Australia between 2016 and 2017. Prevalence costing methods were used and reported in 2017 US dollar (USD) purchasing power parity (PPP).

Wed, 02/01/2023 - 08:42

The Burden of Spinal Muscular Atrophy on Informal Caregivers

Spinal muscular atrophy (SMA) is one of the most common severe hereditary diseases of infancy and early childhood. The progression of this illness causes a high degree of disability; hence, a significant burden is experienced by individuals with this disease and their families. We analyzed the time taken to care for patients suffering from SMA in European countries and the burden on their informal caregivers. We designed a cross-sectional study recording data from France, Germany, Spain and the United Kingdom.

Thu, 12/29/2022 - 12:22

Quality of life of children with spinal muscular atrophy and their caregivers from the perspective of caregivers: a Chinese cross-sectional study

Background: Spinal muscular atrophy (SMA) is an autosomal-recessive motor neuron disease leading to dysfunction of multiple organs. SMA can impair the quality of life (QoL) of patients and family. We aimed to evaluate the QoL of children with SMA and their caregivers and to identify the factors associated with QoL in a cross-sectional study conducted in China. Methods: We recruited 101 children aged 0–17 years with SMA and their caregivers from a children’s hospital in China. Twenty-six children had type I SMA, 56 type II and 19 type III.

Fri, 06/17/2022 - 15:25

The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs

Aim: This study aims to reveal the problems faced by families of children with spinal muscular atrophy (SMA), by evaluating their care burden, needs, and expectations. Materials and Methods: The participants were the primary caregivers of 34 children between the ages of 0 and 18 years diagnosed with SMA. Thirteen children were diagnosed with type 1, 13 children with type 2 and 8 children with type 3 SMA. Data on the medical history, functional levels of the participants, and the characteristics of families were collected.

Sat, 01/15/2022 - 13:55