Spinal Muscular Atrophy Type 2 Overview

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Learn About Spinal Muscular Atrophy Type 2

What is the definition of Spinal Muscular Atrophy Type 2?
Spinal muscular atrophy type 2 (SMA2) is a genetic neuromuscular disorder that affects the nerve cells that control voluntary muscles (motor neurons). Babies with SMA2 can sit without support, however, they cannot stand or walk independently. Feeding and breathing problems may also develop. SMA2 is caused by changes (pathogenic variants also called genetic changes) in the SMN1 gene and is inherited in an autosomal recessive manner. Diagnosis of SMA2 is suspected by symptoms and confirmed by genetic testing.
What are the alternative names for Spinal Muscular Atrophy Type 2?
  • Spinal muscular atrophy type 2
  • Dubowitz disease
  • Muscular atrophy, spinal, infantile chronic form
  • Muscular atrophy, spinal, intermediate type
  • SMA II
  • SMA2
  • Spinal muscular atrophy type II
Who are the top Spinal Muscular Atrophy Type 2 Local Doctors?
Elite in Spinal Muscular Atrophy Type 2
Dr. Richard S. Finkel
Pediatric Neurology | Pediatrics | Neurology
Elite in Spinal Muscular Atrophy Type 2
Dr. Richard S. Finkel
Pediatric Neurology | Pediatrics | Neurology
262 Danny Thomas Pl, 
Memphis, TN 
888-226-4343
Languages Spoken:
English
See accepted insurances
Accepting New Patients
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Richard Finkel is a Pediatric Neurologist and a Pediatrics provider in Memphis, Tennessee. Dr. Finkel is rated as an Elite provider by MediFind in the treatment of Spinal Muscular Atrophy Type 2. His top areas of expertise are Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis, Spinal Muscular Atrophy Type 3, and Spinal Muscular Atrophy Type 2. Dr. Finkel is currently accepting new patients.

Elite in Spinal Muscular Atrophy Type 2
Dr. Basil T. Darras
Pediatric Neurology | Medical Genetics | Pediatrics
Elite in Spinal Muscular Atrophy Type 2
Dr. Basil T. Darras
Pediatric Neurology | Medical Genetics | Pediatrics

C H Neurology Foundation Inc

300 Longwood Ave, 
Boston, MA 
617-355-6000
Languages Spoken:
English
See accepted insurances
Accepting New Patients

Basil Darras is a Pediatric Neurologist and a Medical Genetics provider in Boston, Massachusetts. Dr. Darras is rated as an Elite provider by MediFind in the treatment of Spinal Muscular Atrophy Type 2. His top areas of expertise are Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 1, and Gastrostomy. Dr. Darras is currently accepting new patients.

 
 
 
 
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Elite in Spinal Muscular Atrophy Type 2
Francesco Muntoni
Elite in Spinal Muscular Atrophy Type 2
Francesco Muntoni
Great Ormond Street Hospital Biomedical Research Centre, 
London, ENG, GB 

Francesco Muntoni practices in London, United Kingdom. Mr. Muntoni is rated as an Elite expert by MediFind in the treatment of Spinal Muscular Atrophy Type 2. His top areas of expertise are Becker Muscular Dystrophy, Duchenne Muscular Dystrophy, Primary Lateral Sclerosis, Spinal Muscular Atrophy (SMA), and Gastrostomy.

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What are the latest Spinal Muscular Atrophy Type 2 Clinical Trials?
Analysis of Mobility in the Bath for Infants Affected With Spinal Muscular Atrophy
Analysis of Mobility in the Bath for Infants Affected With Spinal Muscular Atrophy
Enrollment Status: Recruiting
Publish Date: November 21, 2025
Intervention Type: Other
Study Phase: Not Applicable

Summary: Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death. With emerging therapies, a need arises to enhance motor function assessment in severely hypotonic infants (SMA type 1) as traditional scales on examination tables lack completeness due to gravity's influence. The study team has de...

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A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
Enrollment Status: Recruiting
Publish Date: November 19, 2025
Intervention Type: Drug
Study Phase: Phase 2

Summary: This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score...

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Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center