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Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

by BNS Staff | Jan 26, 2021 | Just Published, News, Pulmonologist News, slider

Spinraza (nusinersen) effectively preserves respiratory muscle strength in addition to motor function in children with spinal muscular atrophy (SMA) type 2, according to a real-life, small study in France. Larger studies are needed to confirm these findings, and to...
Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show

Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show

by BNS Staff | Dec 10, 2020 | Just Published, slider

Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 who were unable to walk, according to final results from an extension Phase 2 clinical trial....
4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds

4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds

by BNS Staff | Oct 8, 2020 | Just Published, News, Orthopedics News, slider

Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength of spinal muscular atrophy (SMA) type 3 patients who are able to walk, according to results from a small Phase 2/3 clinical trial.  Findings from...
FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA

FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA

by BNS Staff | Sep 30, 2020 | Just Published, News, Orthopedics News, Pediatricians News, Physical Therapists News, slider

The U.S. Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to SRK-015, an investigational treatment for spinal muscular atrophy (SMA), its developer, Scholar Rock, announced. This designation is given to therapies that are...
Nerve-muscle Communication Molecule May Be SBMA Therapeutic Target, Study Says

Nerve-muscle Communication Molecule May Be SBMA Therapeutic Target, Study Says

by BNS Staff | Sep 24, 2020 | Genetic Counselors News, HCP, Just Published, Neurology News, Orthopedics News, slider

Increasing the levels of brain-derived neurotrophic factor (BDNF) — a molecule involved in the communication between nerve cells and muscle — specifically in muscle delayed symptom onset and slowed disease progression in a mouse model of spinal and bulbar muscular...
Muscle and Combo Therapies Likely Next Focus for SMA, Experts Say

Muscle and Combo Therapies Likely Next Focus for SMA, Experts Say

by BNS Staff | Sep 10, 2020 | Just Published, Neurology News, News, Orthopedics News, Pediatricians News, slider

Treatment combinations, including future muscle-targeted therapies, are part of what likely lies ahead for spinal muscular atrophy (SMA), according to experts in the SMA community. In separate interviews with SMA News Today, Richard Finkel and Laurent Servais,...
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SMA – Core Topics

  • What is SMA?
  • Types of SMA
    • Spinal Muscular Atrophy Type 0
    • Spinal Muscular Atrophy Type 1
      • SMA Type 1 Subtypes
    • Spinal Muscular Atrophy Type 2
      • SMA Type 2 Subtypes
    • Spinal Muscular Atrophy Type 3
      • SMA Type 3 Subtypes
    • Spinal Muscular Atrophy Type 4
    • Rare Forms of SMA
    • SMN1-negative variants resembling Adult Onset SMA
    • SMN1-negative variants resembling Infantile Onset SMA
    • SMN1-negative variants resembling Childhood Onset SMA
    • Pre-Symptomatic Intervention 
  • Genetics and Epidemiology
    • Genetic Counseling
    • Genetic Screening
    • The Role of MicroRNA
    • MUNI Biomarker for SMA
  • Signs and Symptoms
    • Pain
    • Weakness and Hypotonia
  • Comorbid Conditions
    • Abnormal Fatty Acid Oxidation in SMA
    • Cardiac Disease
    • Enuresis and Urinary Incontinence
    • Eosinophilic Oesophagitis in SMA
    • Pancreatic Dysfunction
    • SMA Associated with Progressive Myoclonic Epilepsy
    • Sleep Disorders in SMA
  • Diagnosis
    • Biomarkers
    • Electromyography
    • Muscle Biopsy
    • MRI
  • Differential Diagnosis
    • Prenatal/Neonatal Onset
    • Childhood Onset
    • Adult Onset
  • Disease-Modifying Therapy
    • SPINRAZA (nusinersen)
      • Administering Nusinersen
      • Alternate Nusinersen Administration Strategies
      • How to Administer Nusinersen in Adolescents 
      • Nusinersen: What is Still Unclear
      • Patient and Parent Perspectives on Nusinersen
    • ZOLGENSMA (AVXS-101)
      • Administering Zolgensma
      • Zolgensma: Caregiver Counseling
    • EVRYSDI (Risdiplam)
    • Treatments in Development
      • Branaplam
      • Olesoxime
      • Reldesemtiv
      • Salbutamol
      • Valproic Acid
      • Combined Drug Therapy
      • Disease Modifying Therapies in Spinal Muscular Atrophy: Preclinical Products
  • Testing
    • Meaningful Change in SMA
  • Orthopedic Surgery
    • Chest Deformity, Hip Instability, Contractures, and Fractures
    • Contractures
    • Fractures
    • Scoliosis
    • Scoliosis Surgery
  • Discussing Clinical Trials
  • Respiratory Care
    • Airway Clearance
    • Breathing Difficulty
    • In the Acutely Ill Child
    • In the Well Child
    • Intubation and Extubation 
    • Oxygenation and Breathing 
    • Practical Guidance for Breathing Problems
    • Perioperative Considerations
    • Retained Secretions and Aspiration
  • Physical Therapy
  • Home Modifications
  • Feeding, GERD, and GI Issues
    • Gastrointestinal Access
    • GERD
    • In SMA Patients Who Can Sit Up
    • In SMA Patients Who Cannot Sit Up
  • Delayed Growth
  • School and Educational Concerns
    • Awareness and Attitudes
  • Maximizing Quality of Life
  • Assistive Devices
    • Oxygenation and Breathing 
    • Mobility Devices
    • Braces
    • Feeding
  • Speech Therapy
  • Occupational Therapy
  • Nutrition
    • Assessment of Growth and Growth Delay
    • Chewing and Swallowing Problems
    • Fluids and Hydration
    • Nutrition and Supplementation
  • Medical Transportation and Hospitalization
    • Hospitalization for SMA Patients
  • Pain Management
  • Patient & Caregiver Concerns
    • Caregiver Support
    • Effects on Home, School, and Leisure
    • Maximizing Quality of Life
    • Psychological Support
    • What Living with SMA is Like: A Qualitative Research Perspective
  • Prognosis
  • Palliative Care

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SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.