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Spinal Muscular Atrophy (SMA)

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We are partnering with people living with spinal muscular atrophy (SMA) and patient organisations to explore multiple approaches to treatment that help improve lives.

Challenge

SMA is an autosomal recessive genetic disorder caused by reduced levels of SMN protein throughout the body, resulting from mutations in the survival motor neuron-1 (SMN1) gene. It affects approximately 1 in 10,000 babies born worldwide each year and is the most common genetic cause of death in infants.

It is a disease that robs people of their physical strength by affecting motor nerve cells, taking away the ability to walk, eat and even breathe.

Focus

We have been inspired by the SMA community throughout our ten-year history of research and investigation into the SMA. Working together, we continue to strive for further understanding and progression of the science behind SMA.

Stories of

Spinal Muscular Atrophy

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    How adult SMA patients improve access to care and treatment

    Three letters – SMA – tell countless personal stories. Stories of resilience, as adults living with spinal muscular atrophy (SMA) navigate everyday life and overcome challenges to access care and treatment.

  • A woman in a purple sweater sits in an electric wheelchair, positioned near a glass enclosure. There are wooden bench seats, cushions, and hanging plants. She appears thoughtful.
    Series

    What makes you, you: Seda's story

    Seda lives with spinal muscular atrophy (SMA). She takes us through a day in her life, sharing how her advocacy work, hobbies and passions make her unique.

The strength and resolve of the SMA community have continually inspired us.

Dr Levi Garraway

Roche’s Chief Medical Officer and Head of Global Product Development

Downloads

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Download Celebrating 10 years of partnership in SMA

PDF

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Download Spinal Muscular Atrophy

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Partnering with Pharma

Innovation

At Roche Pharma Partnering we are passionate about science: no matter where it comes from. Through partnerships we can build a better future together with medicines that make a real difference to patients.

Clinical trials

Innovation

Clinical trials are essential to determine whether new drugs or diagnostics are safe and effective when used to diagnose or treat people.