Learn About Spinal Muscular Atrophy Type 3

What is the definition of Spinal Muscular Atrophy Type 3?
Spinal muscular atrophy type 3 (SMA3) is a mild form of an inherited (genetic) neuromuscular disease characterized by decreased muscle tone and weakness (atrophy). Spinal muscular atrophy type 3 is caused by a degeneration of the lower motor neurons in the brain stem and spinal cord that control muscle movement. Spinal muscular atrophy type 3 mainly affects infants and children.
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What are the symptoms of Spinal Muscular Atrophy Type 3?
Symptoms of spinal muscular atrophy type 3 usually appear after the first 18 months of life but can also have a later onset in early adulthood. Symptoms of spinal muscular atrophy include mild muscle weakness, difficulty walking, running, and climbing up and down stairs, and frequent respiratory infections.
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What are the current treatments for Spinal Muscular Atrophy Type 3?
There is currently no cure for spinal muscular atrophy type 3. Promising new treatments include disease-modifying therapies that stimulate the production of the spinal motor neuron protein (SMP), such as intrathecal (injected into spinal canal) Nusinersin (Spinraza) and oral risdaplam (Evrysdi). Other treatments for spinal muscular atrophy type 3 are mainly focused on alleviating symptoms and improving quality of life and may include physical therapy; occupational therapy; and the use of assistive devices, such as braces crutches, walkers and wheelchairs.
Who are the top Spinal Muscular Atrophy Type 3 Local Doctors?
Elite
Highly rated in
42
conditions

Università Cattolica Del Sacro Cuore

Rome, IT 

Eugenio Mercuri is in Rome, Italy. Mercuri is rated as an Elite expert by MediFind in the treatment of Spinal Muscular Atrophy Type 3. He is also highly rated in 42 other conditions, according to our data. His top areas of expertise are Spinal Muscular Atrophy SMA, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Primary Lateral Sclerosis.

Elite
Highly rated in
16
conditions

Università Cattolica Del Sacro Cuore

Rome, IT 00168

Marika Pane is in Rome, Italy. Pane is rated as an Elite expert by MediFind in the treatment of Spinal Muscular Atrophy Type 3. She is also highly rated in 16 other conditions, according to our data. Her top areas of expertise are Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 1, and Becker Muscular Dystrophy.

 
 
 
 
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Elite
Highly rated in
22
conditions
Neurology
Pediatrics

Boston Childrens Hospital

300 Longwood Ave 
Boston, MA 2115

Basil Darras is a Neurologist and a Pediatrics doctor in Boston, Massachusetts. Dr. Darras has been practicing medicine for over 45 years and is rated as an Elite doctor by MediFind in the treatment of Spinal Muscular Atrophy Type 3. He is also highly rated in 22 other conditions, according to our data. His top areas of expertise are Spinal Muscular Atrophy SMA, Spinal Muscular Atrophy Type 1, Primary Lateral Sclerosis, and Spinal Muscular Atrophy Type 3. He is licensed to treat patients in Massachusetts. Dr. Darras is currently accepting new patients.

What are the latest Spinal Muscular Atrophy Type 3 Clinical Trials?
Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients
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A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy
What are the Latest Advances for Spinal Muscular Atrophy Type 3?
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.
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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.