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Spinal muscular atrophy (SMA) is a genetic, progressive, and often terminal rare disease that affects an individual’s ability to walk, eat, and, ultimately, breathe. Debilitating and often fatal, SMA affects approximately one in 10,000 live births and is a leading genetic cause of death among infants.
SMA impacts individuals across a range of ages – from infants and children to teens and adults – with varying levels of severity. Newborns and infants can develop infantile-onset SMA, the most severe form of the disease, which may lead to paralysis and prevent infants from performing the basic functions of life, such as swallowing or holding up their heads. Later-onset SMA is more common among children, teens and adults, who may experience significant muscle weakness and disability, such as the inability to stand or walk independently.
Individuals with two copies of the SMN2 gene, a gene which also produces the survival motor neuron (SMN) protein, are most likely to develop infantile-onset SMA (also known as Type 1), while those with three or four copies are most likely to develop later-onset SMA (Types 2 and 3).
Therapy for SMA
Previously, individuals with SMA and their families had no treatment options. This changed in December 2016 when Biogen received U.S. Food and Drug Administration (FDA) approval for the first therapy approved to treat SMA. The therapy has since been approved in over 40 countries, and has helped thousands of patients around the world to date. Availability in the European Union will vary by country, per local reimbursement and access pathways.
We continue to work with health care professionals, government agencies, policymakers, and advocacy groups to be able to provide access to this new therapy as quickly as possible to as many who may benefit.
Biogen is dedicated to enhancing the lives of those with SMA and their families through ongoing research, removing barriers to access and providing support programs. In December 2017, Biogen launched a new collaboration with Ionis to identify new therapeutic options – specifically, new antisense oligonucleotide (ASO) candidates, which are designed to treat SMA – for individuals afflicted with the debilitating disease.
Please review Biogen’s corporate expanded access program (EAP) principles and criteria. Detailed information on the EAP for eligible individuals with infantile-onset (type 1) SMA, including eligibility criteria and participating sites, is posted on clinicaltrials.gov.
Patients who are interested in more information on this process should contact Biogen's Patient Center.
Inspired by the families whose lives have been touched by SMA, we worked closely with the SMA community to create a disease education program called Together in SMA.
Together in SMA provides information and resources for individuals with SMA and their caregivers. It is our hope that insights gained from this program will help families better navigate living with SMA.
Video: Danyelle’s Story – Raising Two Children with Spinal Muscular Atrophy
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