Biogen Presents New Data Regarding Adults and Infants with SMA

Biogen has announced it will present new data illustrating the rapidly progressive nature of spinal muscular atrophy (SMA) in adults, adolescents and older children at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida (April 13-17, 2019). Other Biogen presentations will highlight the benefits of pre-symptomatic treatment with data from the Nurture study, part of the Spinraza global clinical trial program, and findings on the role of neurofilament as a potential biomarker for predicting motor function in SMA.

Biogen’s presentations on SMA and Spinraza include:

  • Mortality Among a Large, Age-Diverse Population of Patients With Spinal Muscular Atrophy (SMA): A U.S. Epidemiologic Study – Poster 98 – Tuesday, April 16, 6:00 pm-8:00 pm ET
  • Characteristics of Spinal Muscular Atrophy (SMA) Patients in the neuroMuscular ObserVational Research (MOVR) Data Hub – Poster 100 – Tuesday, April 16, 6:00 pm-8:00 pm ET
  • Phosphorylated Neurofilament Heavy Chain (pNF-H) and Motor Function Achievement in Nusinersen-Treated Individuals With Spinal Muscular Atrophy (SMA) – Poster 172 – Tuesday, April 16, 6:00 pm-8:00 pm ET
  • Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy: Interim Results From the Phase 2 NURTURE Study – Poster 174 – Tuesday, April 16, 6:00 pm-8:00 pm ET {Encore presentation}
  • Symptoms and Complications Among Later Childhood, Adolescent and Adult Spinal Muscular Atrophy Patients: A Natural History Study within U.S. Hospitals – Poster Number 206 – Tuesday, April 16, 6:00 pm-8:00 pm ET

Key findings from these presentations include:

  • Adults with later-onset spinal muscular atrophy (SMA) who are not being treated show increased rates of hospital admissions, clinical interventions and mortality, indicating an unmet medical need
  • Infants with pre-symptomatic SMA treated with Spinraza achieve motor milestones that are more consistent with normal development, with 100 percent sitting independently as reported in the NURTURE study
  • Additional data show that neurofilament, a protein that helps brain neurons maintain their structure, could be a biomarker for SMA and may predict improvement in motor function and disease activity

Additional findings and new data are expected in these presentations. Please check back for further updates.

Cure SMA Provides Seed Funding for Spinraza

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach behind SPINRAZA. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.

For More Information

To learn more about Spinraza please visit or the links below:

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