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CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment

by Marta Figueiredo | Mar 3, 2021 | News

Evrysdi (risdiplam) has been recommended for approval in the European Union (EU) to treat spinal muscular atrophy (SMA) patients, ages 2 months and older, with a clinical diagnosis of type 1, 2, or 3, or with one to four copies of the SMN2 “backup” gene. “Our close...
Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1

Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1

by Marta Figueiredo | Mar 1, 2021 | News

Genentech’s Evrysdi (risdiplam) improved survival in infants with spinal muscular atrophy (SMA) type 1, and allowed them to achieve developmental milestones not usually seen in the natural course of the disease, according to one-year data from the dose-finding part of...
Brain Involvement in SMA Type 1 Still Poorly Understood, Review Finds

Brain Involvement in SMA Type 1 Still Poorly Understood, Review Finds

by Marta Figueiredo | Feb 22, 2021 | News

Children with spinal muscular atrophy (SMA) type 1 may have neurodegeneration in several areas of the brain, with impaired cognitive function and profound speech difficulties, according to a review study. However, brain involvement in this severe type of SMA is...
New SMN-boosting Molecule Shows Promise as Add-on Therapy

New SMN-boosting Molecule Shows Promise as Add-on Therapy

by Marta Figueiredo | Feb 17, 2021 | News

Targeting a specific region of an intermediate molecule generated from the SMN2 gene increased the levels of functional SMN — the protein lacking in spinal muscular atrophy (SMA) — in cells derived from SMA patients, a study shows. In addition, combining this approach...
Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients

by Marta Figueiredo | Feb 16, 2021 | News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare...
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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.