Listen to why the Clinical Trials Finder matters, from our Youth Group volunteer Orestis from Greece. 🥰 🔗 Access the website here: http://bit.ly/4gBNl29  🔗 Q&A subsite: http://bit.ly/46k2PVc  #SMA #PatientAdvocacy #weareone

🎉 The Clinical Trials Finder is LIVE! Dr. Nicole Gusset, CEO of SMA Europe, shares: “We know from our community that many people living with SMA want to participate in research but face barriers in accessing clear and timely information.”  🔗 http://bit.ly/4gBNl29 #SMA #research #weareone

🎉 The Clinical Trials Finder is LIVE! Nadya Karachevtseva, Patient Advocate & Volunteer of SMA Europe, shares:“This tool is more than a website; it's a bridge to the future. It turns our hope for better treatments into actionable progress, together.”   🔗 http://bit.ly/4gBNl29 #research #weareone

⏳ Early Bird registration closes Oct 6! Join us at the 5th International Scientific Congress on SMA in Budapest, 11–14 Mar 2026. Don’t miss your chance to connect with scientists, clinicians & researchers worldwide!  🔬 Register: https://congress.sma-europe.eu/ #WeAreOne #SMA #raredisease

♿🎉 Our Accessibility Guidebook is live! Plan your SMA Congress visit with ease — venue info, travel tips, on-site support & more. Questions? Contact Timi: [email protected] 🔗 https://congress.sma-europe.eu/ #spinalmuscularatrophy #accessibility #congress #budapest #weareone

Welcome to SMA Europe Youth SMArt TV. This is Episode 6: First Job Experience with SMA. In this episode, we explore the journey of entering the working world while living with SMA. Watch it here: youtu.be/wRZeg_YHxDg?... #WeAreOne #SMArtTV #SMAEurope #InclusionMatters #YouthVoices

📣 5th International Scientific Congress on SMA. Only 3 days left! Submit your abstract and register! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

Today, Biogen announced that the FDA has issued a decision for its supplemental New Drug Application for a high-dose regimen of nusinersen in the treatment of SMA.https://www.sma-europe.eu/news/community-update-fda-decision-on-the-high-dose-regimen-of-nusinersen-in-the-treatment-of-sma?ref=%2Fnews

Scholar Rock announced that the U.S. (FDA) has delayed approval of the Biologics License Application (BLA) for apitegromab in the treatment of people living with SMA https://www.sma-europe.eu/news/community-update-fda-decision-on-the-approval-of-scholar-rock-s-apitegromab?ref=%2Fnews

Our 1st International Clinical and Care Symposium on SMA brought together almost 200 participants. From powerful patient testimonies to compelling science, the message was clear: "Focus cannot only be on medicines alone, but also on care." Together, #weareone #spinalmuscularatrophy #smacaresymposium

Abstract submission deadline is extended!  📣 5th International Scientific Congress on SMA: submit your abstract now! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

Our 1st International Clinical and Care Symposium on SMA, held on 29-30 August 2025, in Milan, Italy, was a great success! 🥳 This was a first step. And more will follow.  Stay tuned. Together, #weareone  #spinalmuscularatrophy #smacaresymposium

Our 1st International Clinical Care Symposium on SMA showed what happens when patients, caregivers, and clinicians come together: - Knowledge shared - Gaps identified - Solutions set in motion Together, #weareone  #spinalmuscularatrophy #smacaresymposium #smacommunity

Only one week left 📣 5th International Scientific Congress on SMA: submit your abstract now! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

Call for workshops – 5th International Scientific Congress on SMA! We’re inviting researchers, clinicians, and innovators to submit abstracts and host interactive workshops as part of our scientific programme. Join Us! https://congress.sma-europe.eu/abstract-submission-guidelines-for-workshops/

A great 🌎 international collaboration with the Viero (Trento), Cousin (Edinburgh) and Groen (Utrecht; @ewout.bsky.social) labs, led by Federica, Nikky and Anna (@dibbouk.bsky.social). Very grateful to all funders for their support, including the EU Horizon 2020 Network (SMABEYOND) and SMA Europe 🙏

🚨 New #SMA paper from the lab just published in @jci-insight.bsky.social: Prenatal SMN-dependent defects in translation uncover reversible primary cilia phenotypes in spinal muscular atrophy insight.jci.org/articles/vie...

We are very excited to share the article about our 2025 Awareness Campaign, "Connecting the Dots" written by Jacob Harney for SMA News Today. https://smanewstoday.com/news/sma-awareness-campaign-aims-connect-research-patient-needs/ Together #weareone #spinalmuscularatrophy #smaawareness #smaresearch

Call for workshops – 5th International Scientific Congress on SMA! We’re inviting researchers, clinicians, and innovators to submit abstracts and host interactive workshops as part of our scientific programme. Join Us! https://congress.sma-europe.eu/abstract-submission-guidelines-for-workshops/

We want to share an important update regarding the application to include Risdiplam on the World Health Organization’s Model List of Essential Medicines (EML) and Essential Medicines for Children (EMLc). You can find more details on the WHO website here: http://bit.ly/4mL5lJF #spinalmuscularatrophy

Our 1st International Clinical and Care Symposium on SMA, held on 29-30 August 2025, in Milan, Italy, was a great success! This was a first step. And more will follow. Stay tuned. THANK YOU ALL for making history with us. #spinalmuscularatrophy #smacaresymposium #smacare #patientadvocacy

📣 5th International Scientific Congress on SMA: submit your abstract and register! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

Our CEO Dr. Nicole Gusset is featured in Rare Revolution Magazine’s “Beyond the Breakthroughs”! She shares insights on SMA treatments, the power of community & our ongoing journey. Together, we keep driving progress. 🔗 bit.ly/4lO5Tx6 #SMA #WeAreOne #SMAEurope

Thank you all who watched and engaged in our campaign! If you want to keep in touch, you can subscribe to our monthly newsletter here: https://www.sma-europe.eu/ And if you want to support the research projects we fund, you can click here: https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Jakub, the patient expert, and Sorana, the researcher: "Investigating Calcium-induced mitochondrial dysfunction in zebrafish and iPSC models of SMA." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

⏰ Last minute registrations! Be part of SMA Europe’s 1st Clinical Care Symposium, 29–30 Aug in Milan. Let’s improve emergency care in SMA. 🔗 https://www.sma-europe.eu/sma-care-symposium #SMAEurope #SMACareSymposium #SMACommunity

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Jakub, the patient expert, and Sorana, the researcher: "Investigating Calcium-induced mitochondrial dysfunction in zebrafish and iPSC models of SMA." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

📣 5th International Scientific Congress on SMA: submit your abstract and register! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

🗓️ 1 week to go! Register for SMA Europe’s 1st Clinical Care Symposium on 29–30 Aug in Milan. The event is accredited for continuing medical education for healthcare professionals. Focus: Medical Emergencies in SMA.  🔗 https://www.sma-europe.eu/sma-care-symposium #SMAEurope #SMACareSymposium

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Seda, the patient expert, and Gabrielle, the researcher: "Leveraging SMN role in translation to develop the next-gen of biomarkers for SMA." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Seda, the patient expert, and Gabrielle, the researcher: "Leveraging SMN role in translation to develop the next-gen of biomarkers for SMA." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🔬This SMA Awareness Month, we are Connecting the Dots! Listen to Christian, the patient expert, and Nathalie, the researcher: "Skeletal Muscle Stem Cells as untapped therapeutic targets for SMA long-term treatment (SATSMA)." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🗓️ 2 weeks to go! Register for SMA Europe’s 1st Clinical Care Symposium on 29–30 Aug in Milan. Focus: Medical Emergencies in SMA. Deadline: 15 Aug. 🔗 https://www.sma-europe.eu/sma-care-symposium #SMAEurope #SMACareSymposium #SMAResearch

🔬This SMA Awareness Month, we are Connecting the Dots! Listen to Christian, the patient expert, and Nathalie, the researcher: "Skeletal Muscle Stem Cells as untapped therapeutic targets for SMA long-term treatment (SATSMA)." https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Simona, the patient expert, and Simon, the researcher: "Are microvascular defects relevant in Spinal Muscular Atrophy?: Characterisation of the mouse model." youtu.be/o1R5h3j1lLE #SMAAwareness #ConnectingTheDots

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Simona, the patient expert, and Simon, the researcher: "Are microvascular defects relevant in Spinal Muscular Atrophy?: Characterisation of the mouse model." youtube.com/shorts/R85PI... #SMAAwareness #ConnectingTheDots

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Christiana, the patient expert, and Morgan, the researcher: "Deciphering the Molecular Landscape of Neuromuscular Development in Spinal Muscular Atrophy." 🔗 https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🔬 This SMA Awareness Month, we are Connecting the Dots! Listen to Christiana, the patient expert, and Morgan, the researcher: "Deciphering the Molecular Landscape of Neuromuscular Development in Spinal Muscular Atrophy." 🔗 https://www.sma-europe.eu/donate #WeAreOne #SMAAwareness #ConnectingTheDots

🔬This Spinal Muscular Atrophy Awareness Month, from SMA Europe, we are Connecting the Dots! Research in SMA is vital. Did you ever wonder how research can have a real impact on the lives of people who live with SMA? Only together, WeAreOne #SMAAwareness #ConnectingTheDots #SpinalMuscularAtrophy

📣 5th International Scientific Congress on SMA: submit your abstract and register! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

Children and adults are dying not because SMA is untreatable, but because systems fail in emergencies. Let’s not wait for another preventable loss. Join the 1st International Clinical and Care Symposium In-person and online registrations are available: https://www.sma-europe.eu/sma-care-symposium

Are you a healthcare professional treating SMA patients? Join international experts, patient leaders, and caregivers to tackle the most urgent challenges in rare disease care. 1st International Clinical and Care Symposium. In-person and online: https://www.sma-europe.eu/sma-care-symposium

"In that moment, I knew my child was in danger, but the ER team had no idea what to do with an SMA patient." Caregivers should not have to educate the ER in a crisis. Join us at the 1st International Clinical and Care Symposium, in person or online: https://www.sma-europe.eu/sma-care-symposium

SMA Europe joined the Cure SMA Conference, joining key talks on SMA research. Big thanks to Cure SMA for a great event! Let’s keep the momentum going: 🔗 Milan, 29–30 August 2025: sma-europe.eu/sma-care-symposium 🔗 Budapest, March 2026: https://congress.sma-europe.eu/ #WeAreOne #SMA #SMACommunity

Our CEO, Nicole Gusset, co-authored the “Navigating Patient-Focused Innovation: One Company’s Journey with the SMA Community”, published by DIA Global Forum. Read it here: https://bit.ly/40VntHN #SpinalMuscularAtrophy #PatientAdvocacy #RareDisease

People living with SMA repeatedly end up in life-threatening metabolic crises, and this is preventable. Let us exchange our knowledge, and build faster, smarter emergency response pathways. Join us at the 1st International Clinical and Care Symposium https://www.sma-europe.eu/sma-care-symposium

📣 5th International Scientific Congress on SMA: submit your abstract and register! 🔬 The event will take place from 11th to 14th March 2026 in Budapest, Hungary. Visit https://congress.sma-europe.eu/ to learn more #spinalmuscularatrophy #research #raredisease

"Choking is rare for me. But when it happens and no one is there to help with tools like the Cough Assist, I panic. We have tried many times to set up an emergency plan, but still have none. Join us at the 1st International Clinical and Care Symposium: https://www.sma-europe.eu/sma-care-symposium

📣 SMA Europe’s Annual Report 2024 is here! Discover our key projects, progress, and the people behind them — all working for a better world for everyone living with SMA. 💜 🔗 https://heyzine.com/flip-book/94ea793aba.html#page/8 #WeAreOne #SMAEurope #spinalmuscularatrophy #raredisease #patientadvocacy

What’s more urgent than saving a life? 📣 Today, children and adults with SMA are still dying unnecessarily during emergencies because healthcare systems lack the knowledge on how to respond. Join us at our 1st International Clinical Care Symposium: https://www.sma-europe.eu/sma-care-symposium