Advocacy

SMA Europe campaigns to improve the quality of life of people who live with SMA and to bring effective therapies to patients in a timely and sustainable way, as well as to encourage optimal patient care.

We do this in a variety of ways:

• We inform on patients’ expectations,
• We inform on the burden of living with the disease,
• We inform on the potential economic and societal benefits of stabilising or improving health conditions of the SMA population
• We provide important information that will inform the development of drugs which have the most meaningful effects to patients

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Our position statements

• Opening a new horizon for children born with SMA – Newborn screening – 31st August 2020
• Zolgensma®, the first gene therapy to treat SMA, receives conditional marketing authorisation in the European Union – May 2020
• COVID-19 & the needs of people living with SMA – April 2020
• EMA recommendation of Zolgensma®: SMA Europe urges stakeholders to work together towards making Zolgensma® widely available across Europe – April 2020
• Access to Therapies for SMA – December 2018
• Patient-centered Outcome Measures (PCOs) – June 2018

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Our activities

• Patient involvement in decision making
• Clinical trials initiatives
• Our European Alliance for newborn screening for SMA