spinal-muscular-atrophy Clinical Trials
A listing of spinal-muscular-atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 67 clinical trials
Electrical Stimulation for the Treatment of Optic Neuropathies
The overall aim of this study is to see whether long-term electrical stimulation with a home-stimulation device works well and is safe for the treatment of open-angle glaucoma. Open-Angle Glaucoma is a disease where the nerves in the back of your eye die off faster than expected regardless of your …
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder. The main questions it aims to answer are: What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb …
Alternating and Direct Current Stimulation for Neuropathic Eye Pain
The goal of this clinical intervention is to test if two forms of transcranial current stimulation, transcranial direct current stimulation (tDCS) or transcranial alternating current stimulation (tACS) can alleviate neuropathic eye pain in a sample of 20 patients. The main aims are: Test if tDCS/tACS can alleviate neuropathic eye pain …
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
Natural History of SMA
This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease registry.
A Study of Spinraza (Nusinersen) Exposure in Pregnant Women With Spinal Muscular Atrophy (SMA) Within Existing SMA Registries
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) …
Neonatal Spinal Muscular Atrophy (SMA) Screening
Parents or legal guardian of neonates who signed agreement will receive SMA screening test if their neonates are affected with SMA. The dried blood spots of routine newborn screening samples will be used to test if neonates have lost 2 copies of SMN1 gene. If neonates have positive SMA screening …
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence …
Spinal Muscular Atrophy Neonatal Screening Program
Spinal muscular atrophy (SMA) is a group of disorders caused by the degeneration of the motor neuron cells of the anterior horn of the spinal cord and, in some subtypes, of the bulbar motor neurons. Almost all cases are genetically determined. Most SMAs are autosomal recessive diseases, caused by homozygous …
French Register of Patients With Spinal Muscular Atrophy
The primary objectives of the study are to obtain clinically meaningful data on survival and outcomes of all the patients with spinal muscular atrophy (SMA) 5q types 1 through 4 (according to international classification), being followed in the reference centers of the disease in France between September 1, 2016 and …
