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Neurologists Confident SMA Treatments Help Youngest Patients Most

Neurologists Confident SMA Treatments Help Youngest Patients Most

by Marta Figueiredo PhD | Jun 17, 2022 | News

Pediatric neurologists’ expectations in terms of treatment benefits are decidedly higher for infants and toddlers with spinal muscular atrophy (SMA) types 1 and 2 than for adolescent patients, a study in Spain shows. Notably, neurologists with low aversion to...
Infants Screened for Zolgensma Should Repeat Antibody Tests

Infants Screened for Zolgensma Should Repeat Antibody Tests

by Marta Figueiredo PhD | Jun 3, 2022 | News

Infants with spinal muscular atrophy (SMA) who are being screened for Zolgensma may present with maternal antibodies against the therapy’s viral carrier — making them ineligible for the therapy — until the age of 8 months, a small study shows. This means that an...
Evrysdi Safe in Type 1, 2 Patients in US Access Program Before Approval

Evrysdi Safe in Type 1, 2 Patients in US Access Program Before Approval

by Marta Figueiredo PhD | May 20, 2022 | News

Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an analysis of...
Zolgensma Safe in Children Heavier Than Those in Trials, Data Show

Zolgensma Safe in Children Heavier Than Those in Trials, Data Show

by Marta Figueiredo PhD | May 18, 2022 | News

Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier group. Notably, 8.5 kilograms was the maximum weight of SMA patients...
#AAN2022 – Spinraza Showing Safety in Children With Needs After Zolgensma

#AAN2022 – Spinraza Showing Safety in Children With Needs After Zolgensma

by Marta Figueiredo PhD | Apr 29, 2022 | News

Treatment with Spinraza (nusinersen) appears safe in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma, early safety data from the first nine RESPOND trial patients show. Motor and lung function were the most...
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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.