SMA Europe

SMA Europe SMA Europe is a non-profit umbrella organisation of SMA patient organisations from across Europe.
(1)

💡 Did you know?87.1% of participants in our EUPESMA survey reported that they would participate in a clinical trial.Amon...
27/11/2025

💡 Did you know?

87.1% of participants in our EUPESMA survey reported that they would participate in a clinical trial.

Among those who have already taken part in a trial:
- 79.4% described their experience as positive
- 20.6% reported a neutral or very negative experience

This highlights that, while many experiences are positive, there are still areas for improvement and unmet needs. Addressing these gaps is essential when designing new patient-centric clinical trials in the future.

📖 Want to learn more?
Read our SMA Europe’s One Goal editorial series publication, "Together We Prioritise: Results from EUPESMA on Clinical Trial Participation" here: https://heyzine.com/flip-book/9c8efc8e88.html

💬 98% of people living with SMA who participated in our EUPESMA survey say: there is a need for new clinical trials!Our ...
25/11/2025

💬 98% of people living with SMA who participated in our EUPESMA survey say: there is a need for new clinical trials!

Our latest One Goal – SMA Europe publication, “Together we prioritise. Results from EUPESMA on Clinical Trial Participation”, shares insights from 851 participants across 51 countries 🌍

✨ Every voice matters. Every perspective counts. These insights will help guide future SMA clinical trials and ensure that patient perspectives are at the centre.

📖 Read the full report: https://heyzine.com/flip-book/9c8efc8e88.html

Breaking News! 📣🧬Today, Novartis announced that the company has received the FDA approval for Itvisma® (onasemnogene abe...
25/11/2025

Breaking News! 📣🧬

Today, Novartis announced that the company has received the FDA approval for Itvisma® (onasemnogene abeparvovec-brve), the intrathecal formulation of onasemnogene abeparvovec, to treat SMA in adult and pediatric patients 2 years of age and older with a confirmed mutation in SMN1.

In Europe, the therapy is currently undergoing a Joint Clinical Assessment, with results expected in mid-2026.

Please find further information here: https://bit.ly/4pzd5Qb

21/11/2025

Workshop registration NOW OPEN!

We are excited to announce that registration for the workshops at the 5th International Scientific Congress on Spinal Muscular Atrophy is open! 🙌
On 11 March 2026, participants will have the opportunity to join two workshop sessions, each offering six parallel workshops covering the latest developments in SMA research, care, and innovation.

🟣 Seats are limited — registration will operate on a first-come, first-served basis.

Register for workshops: https://congress.sma-europe.eu/workshop/

We look forward to welcoming you in Budapest for a day of scientific exchange, collaboration, and discovery. Together working towards a brighter future for everyone living with SMA. 💜

The workshops are kindly supported by ArgenX , Biogen , Novartis , Roche , Scholar Rock.

20/11/2025

📣 We’re very excited to announce the latest publication in SMA Europe’s One Goal editorial series!

🤓 Together We Prioritise: Results from EUPESMA on Clinical Trial Participation

This report draws on patient experience data from the pan-European EUPESMA survey, providing real insights into clinical trial participation and the community’s hopes and expectations for the future.

Our goal: to ensure that patient perspectives directly shape the design and planning of future clinical trials.

📊 Key highlights from this fourth EUPESMA survey:

- Conducted in 51 countries around the world and translated into 14 languages

- Gathers the voices of people living with SMA to understand their clinical trial experiences

- The survey was open to people living with SMA (16 years old or older), and parents and caregivers of people living with SMA, including both people who had participated in clinical trials before and people who had not.

🙌 Read the full publication here: https://heyzine.com/flip-book/9c8efc8e88.html

Together,


This survey was carried out in collaboration with Tilburg University (Netherlands) and supported by the Cure SMA Industry Collaboration

Community Update!Biogen announced that the high dose regimen of nusinersen received a positive opinion from the Committe...
17/11/2025

Community Update!

Biogen announced that the high dose regimen of nusinersen received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

This will now be reviewed by the European Commission, with a final decision on its approval expected in January 2026.

You may read the full press release here: https://investors.biogen.com/news-releases/news-release-details/high-dose-regimen-nusinersen-receives-positive-chmp-opinion

10/11/2025

Today, we celebrate World Science Day for Peace and Development.🧬🌍

Thomas Koed Doktor , PhD, Chair of the Treatment Committee at SMA Europe, reminds us: “Science builds bridges between people, ideas, and possibilities.”

At SMA Europe, we believe that every discovery, every insight, and every act of curiosity can connect us across disciplines and borders, driving patient-centred research in the SMA field.

Let’s keep connecting the dots: for progress, equality, and hope. Together, we shape a future where no one is left behind. 💫

Between 27 and 30 October, our SMA Europe Staff Team gathered in Freiburg, Germany for a few days of team-building and r...
07/11/2025

Between 27 and 30 October, our SMA Europe Staff Team gathered in Freiburg, Germany for a few days of team-building and reflection.

It was a wonderful opportunity to review our ongoing projects and initiatives, contribute to the planning of 2026, and strengthen our shared vision for the future.

Moments like these remind us that behind every impactful project is a team united by purpose, trust, and a lot of motivation.

All together. One goal. 💫

04/11/2025

Have you explored our Clinical Trials Finder yet?

"With just a few inputs, you can find every clinical trial relevant to you — not only in Europe, but across the world.” Nicole from our Youth Group in South Africa.

🔗 Access the website here: http://bit.ly/4gBNl29
🔗 Q&A subsite: http://bit.ly/46k2PVc

28/10/2025

Let’s make sure everyone in our SMA community knows about the Clinical Trials Finder! 💜

“For us, knowledge really is power. Staying informed about new treatments can make all the difference.” Elise from our Youth Group in The Netherlands.

🔗 Access the website here: http://bit.ly/4gBNl29
🔗 Q&A subsite: http://bit.ly/46k2PVc

22/10/2025

Still haven’t checked our Clinical Trials Finder out? 👀

"It gives me hope. It shows me that science is moving forward and that we have the right to be part of it” Orestis from our Youth Group in Greece.

🔗 Access the website here: http://bit.ly/4gBNl29
🔗 Q&A subsite: http://bit.ly/46k2PVc

New Publication!We are excited to share the latest article about our Clinical Trials Finder, written by SMA News TodayTh...
16/10/2025

New Publication!

We are excited to share the latest article about our Clinical Trials Finder, written by SMA News Today

The article highlights the importance of this new online tool, designed by SMA Europe to help patients, families, and clinicians easily explore ongoing SMA clinical trials across Europe and worldwide.

It presents the tool as “the go-to global resource” for transparent and accessible information on clinical research — offering a centralised, user-friendly platform where the SMA community can search for trials tailored to individual needs and stay informed with real-time updates.

You can read the full text here: http://bit.ly/4nQf8yT (link also in Bio)

🔗 Explore the tool here: http://bit.ly/4gBNl29 (link also in Bio)

We will continue to empower our community with knowledge and tools that connect science and patients. Together

Adresse

Im Moos 4
Freiburg Im Breisgau
79112

Webseite

Benachrichtigungen

Lassen Sie sich von uns eine E-Mail senden und seien Sie der erste der Neuigkeiten und Aktionen von SMA Europe erfährt. Ihre E-Mail-Adresse wird nicht für andere Zwecke verwendet und Sie können sich jederzeit abmelden.

Die Praxis Kontaktieren

Nachricht an SMA Europe senden:

Verknüpfungen

Teilen

Share on Facebook Share on Twitter Share on LinkedIn
Share on Pinterest Share on Reddit Share via Email
Share on WhatsApp Share on Instagram Share on Telegram