View clinical trials related to Muscular Atrophy.
Filter by:This observational study will use new smartwatch technology to continuously and remotely monitor the health of ALS patients and healthy controls over time. This information will be used to develop digital biomarkers for ALS.
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
The goal of this pilot randomized controlled trial is to compare the effects of aquatic therapy versus standard care on paraspinal and gluteal morphology and function in individuals with chronic low back pain.The main questions it aims to answer are: 1. What are the effects of aquatic therapy versus standard care on a) paraspinal and gluteal muscle size, composition (e.g., fatty infiltration) and b) lumbar and gluteal muscle strength in individuals with chronic LBP? 2. Is aquatic therapy more effective than standard care to improve pain, function and psychological factors (e.g., kinesiophobia, catastrophizing, anxiety, and depression)? 3. Is using a digital application "play the pain" feasible to monitor pain levels and the activities that participants used to cope with pain? Participants will be assigned to either the aquatic therapy group or standard care group where they will undergo a 10-week intervention including two 60-minute session per week.
Patients admitted to the hospital often develop functional impairments due to being in bed most of the day. Each day of bedrest leads to significant muscle loss. As a result, many patients become dependent on others or require rehabilitation at a facility to improve mobility and function prior to returning home. Staff in the hospital is limited and often unable to mobilize patients every day while hospitalized. The investigators are testing a new experimental gamified physical therapy exercise software to see if it can be a fun, enjoyable way to help mobilize patients without the assistance of staff. The primary aim of this pilot/proof of concept study is to determine whether gamified physical therapy software can help inpatients exercise within the safety of their own beds and preserve pre-hospitalization function.
The purpose of this study is to assess the impact of 3-days reduced physical activity (<1500 steps/day) with/without 'exercise snacks' (15 chair stands with calf raises every 30 min) on skeletal muscle metabolic health.
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
The primary objectives of the study are to prospectively evaluate pregnancy complications and outcomes in participants with SMA, birth outcomes and adverse effects in infants born to participants with SMA, who were exposed to nusinersen up to 14 months prior to the first day of their last menstrual period (LMP) before conception, 14.5 months before the date of conception, and/or at any time during their pregnancy. The secondary objective of the study is to evaluate pregnancy outcomes in participants with SMA exposed to nusinersen as compared with participants without SMA who were not exposed to nusinersen (e.g., participants from external, general population comparators).
Major breakthroughs in the treatment for Spinal muscular atrophy (SMA) have been recently achieved with various therapeutic approaches that increase full-length SMN protein levels. The variability observed following the advent of commercial availability of Nusinersen for all types of SMA has highlighted the need to identify tools that may allow to predict possible therapeutic responses. The aim of this project is to establish whether an integrated approach using clinical, imaging (muscle MRI) and circulating biomarkers, can provide the possibility to develop a predictive model of therapeutic response to novel therapies for SMA patients. More specifically we wish to establish the correlation between clinical response, different biomarkers indicative of central nervous system efficacy (e.g. determination of neurofilaments levels), and markers that provide evidence of the skeletal muscle response (e.g. serum myostatin and muscle imaging) in different types of SMA
This study will evaluate if Ursolic Acid supplementation may be effective in reducing muscle loss and improving blood sugar control in the SCI community.