Cure MFM13
@curemfm13.bsky.social
A patent-driven charitable project, united by a common cause: to address the challenges of MFM13
Proud to represent CureMFM13 at the #WODC2025 in Amsterdam 🇳🇱!
We presented our poster CureMFM13: Connecting Patients, Research, and Industry”— sharing how collaboration drives progress for #MFM13 and the rare disease community.
#RareDisease #PatientAdvocacy #GeneTherapy
We presented our poster CureMFM13: Connecting Patients, Research, and Industry”— sharing how collaboration drives progress for #MFM13 and the rare disease community.
#RareDisease #PatientAdvocacy #GeneTherapy
November 11, 2025 at 6:02 PM
Proud to represent CureMFM13 at the #WODC2025 in Amsterdam 🇳🇱!
We presented our poster CureMFM13: Connecting Patients, Research, and Industry”— sharing how collaboration drives progress for #MFM13 and the rare disease community.
#RareDisease #PatientAdvocacy #GeneTherapy
We presented our poster CureMFM13: Connecting Patients, Research, and Industry”— sharing how collaboration drives progress for #MFM13 and the rare disease community.
#RareDisease #PatientAdvocacy #GeneTherapy
🎉 Exciting news!
MSc Veronica Marchesi has been admitted to the PhD program in Pharmacological Biomolecular Sciences, Experimental and Clinical at the University of Milan.
We’re thrilled that she will continue her important work on #MFM13, and can’t wait to see all her future successes!
MSc Veronica Marchesi has been admitted to the PhD program in Pharmacological Biomolecular Sciences, Experimental and Clinical at the University of Milan.
We’re thrilled that she will continue her important work on #MFM13, and can’t wait to see all her future successes!
November 3, 2025 at 6:01 PM
🎉 Exciting news!
MSc Veronica Marchesi has been admitted to the PhD program in Pharmacological Biomolecular Sciences, Experimental and Clinical at the University of Milan.
We’re thrilled that she will continue her important work on #MFM13, and can’t wait to see all her future successes!
MSc Veronica Marchesi has been admitted to the PhD program in Pharmacological Biomolecular Sciences, Experimental and Clinical at the University of Milan.
We’re thrilled that she will continue her important work on #MFM13, and can’t wait to see all her future successes!
Great to see Ania Kordala, from @CureMFM13, at WDAD 2025 by Stop Duchenne
Highlights included:
🧬 Prof. Joel Chamberlain on building a mouse model for FSHD
💡 Prof. Agnieszka Łoboda on H₂S donors improving muscle health in DMD mice
Cross-disease science = progress for all
#DMD #FSHD #RareDisease
Highlights included:
🧬 Prof. Joel Chamberlain on building a mouse model for FSHD
💡 Prof. Agnieszka Łoboda on H₂S donors improving muscle health in DMD mice
Cross-disease science = progress for all
#DMD #FSHD #RareDisease
October 31, 2025 at 5:07 PM
Great to see Ania Kordala, from @CureMFM13, at WDAD 2025 by Stop Duchenne
Highlights included:
🧬 Prof. Joel Chamberlain on building a mouse model for FSHD
💡 Prof. Agnieszka Łoboda on H₂S donors improving muscle health in DMD mice
Cross-disease science = progress for all
#DMD #FSHD #RareDisease
Highlights included:
🧬 Prof. Joel Chamberlain on building a mouse model for FSHD
💡 Prof. Agnieszka Łoboda on H₂S donors improving muscle health in DMD mice
Cross-disease science = progress for all
#DMD #FSHD #RareDisease
Update from our visit to PORT Łukasiewicz, Wrocław
We joined the meeting; Models of Collaboration between Patient Advocacy Groups and Scientists.
Met inspiring Patient Advocacy groups
Learned from scientists about new research & testing
Left with fresh ideas for future Cure HSPB8
#RareDisease #MFM13
We joined the meeting; Models of Collaboration between Patient Advocacy Groups and Scientists.
Met inspiring Patient Advocacy groups
Learned from scientists about new research & testing
Left with fresh ideas for future Cure HSPB8
#RareDisease #MFM13
September 26, 2025 at 5:02 PM
Update from our visit to PORT Łukasiewicz, Wrocław
We joined the meeting; Models of Collaboration between Patient Advocacy Groups and Scientists.
Met inspiring Patient Advocacy groups
Learned from scientists about new research & testing
Left with fresh ideas for future Cure HSPB8
#RareDisease #MFM13
We joined the meeting; Models of Collaboration between Patient Advocacy Groups and Scientists.
Met inspiring Patient Advocacy groups
Learned from scientists about new research & testing
Left with fresh ideas for future Cure HSPB8
#RareDisease #MFM13
We’re excited to welcome Vânia as our new Bookkeeper! 🎉
With a Master’s in Financial Auditing and 10+ years of experience in accounting, taxes & financial processes, she’ll help Cure HSPB8 advance its mission and create lasting impact.
With a Master’s in Financial Auditing and 10+ years of experience in accounting, taxes & financial processes, she’ll help Cure HSPB8 advance its mission and create lasting impact.
September 18, 2025 at 5:05 PM
We’re excited to welcome Vânia as our new Bookkeeper! 🎉
With a Master’s in Financial Auditing and 10+ years of experience in accounting, taxes & financial processes, she’ll help Cure HSPB8 advance its mission and create lasting impact.
With a Master’s in Financial Auditing and 10+ years of experience in accounting, taxes & financial processes, she’ll help Cure HSPB8 advance its mission and create lasting impact.
🧬 Help turn rare into cured.
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
September 1, 2025 at 5:05 PM
🧬 Help turn rare into cured.
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
We’re deeply grateful to VoLoFoundation for their renewed support of Cure HSPB8! This crucial funding fuels our fight for a cure, advances research, and strengthens our community. Your continued belief in our mission means everything.
Read more about our collaboration here: buff.ly/xhg6oLm
Read more about our collaboration here: buff.ly/xhg6oLm
July 18, 2025 at 5:04 PM
We’re deeply grateful to VoLoFoundation for their renewed support of Cure HSPB8! This crucial funding fuels our fight for a cure, advances research, and strengthens our community. Your continued belief in our mission means everything.
Read more about our collaboration here: buff.ly/xhg6oLm
Read more about our collaboration here: buff.ly/xhg6oLm
Patient data is one of our most powerful tools. Do you know someone affected by #HSPB8 myopathy? Ask them to join our patient registry: curehspb8.org/families/#Jo...
#PatientRegistry #MyopathyResearch #PatientAdvocacy #Join-The-Registry
#PatientRegistry #MyopathyResearch #PatientAdvocacy #Join-The-Registry
July 7, 2025 at 5:04 PM
Patient data is one of our most powerful tools. Do you know someone affected by #HSPB8 myopathy? Ask them to join our patient registry: curehspb8.org/families/#Jo...
#PatientRegistry #MyopathyResearch #PatientAdvocacy #Join-The-Registry
#PatientRegistry #MyopathyResearch #PatientAdvocacy #Join-The-Registry
🧬 Help turn rare into cured.
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
July 2, 2025 at 5:03 PM
🧬 Help turn rare into cured.
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
Get the latest on HSPB8 research, treatments & patient voices in the Cure HSPB8 newsletter.
Join the movement 👉 curehspb8.org
#CureHSPB8 #RareDisease
Dr. Ania Kordala opened #Genomica in Kraków 🇵🇱 by introducing @CureHSPB8 and speaking on how patients are driving research forward in #ultrarare diseases.
Patient-led advocacy is shaping science and pushing it toward impact.
#RareDisease #Genomics #PatientVoice
Patient-led advocacy is shaping science and pushing it toward impact.
#RareDisease #Genomics #PatientVoice
June 20, 2025 at 5:05 PM
Dr. Ania Kordala opened #Genomica in Kraków 🇵🇱 by introducing @CureHSPB8 and speaking on how patients are driving research forward in #ultrarare diseases.
Patient-led advocacy is shaping science and pushing it toward impact.
#RareDisease #Genomics #PatientVoice
Patient-led advocacy is shaping science and pushing it toward impact.
#RareDisease #Genomics #PatientVoice
🎧 Ep. 3 = Journal Club time!
We dig into HSPB8: how do mutations lead to dHMN, CMT2L & myopathy? Based on Rashed et al.’s new review in IJMS.
🎧 buff.ly/IXA0lo4
🧬 #RareDisease #HSPB8 #Neuromuscular #JournalClub #Podcast
We dig into HSPB8: how do mutations lead to dHMN, CMT2L & myopathy? Based on Rashed et al.’s new review in IJMS.
🎧 buff.ly/IXA0lo4
🧬 #RareDisease #HSPB8 #Neuromuscular #JournalClub #Podcast
June 13, 2025 at 5:04 PM
🎧 Ep. 3 = Journal Club time!
We dig into HSPB8: how do mutations lead to dHMN, CMT2L & myopathy? Based on Rashed et al.’s new review in IJMS.
🎧 buff.ly/IXA0lo4
🧬 #RareDisease #HSPB8 #Neuromuscular #JournalClub #Podcast
We dig into HSPB8: how do mutations lead to dHMN, CMT2L & myopathy? Based on Rashed et al.’s new review in IJMS.
🎧 buff.ly/IXA0lo4
🧬 #RareDisease #HSPB8 #Neuromuscular #JournalClub #Podcast
What are the symptoms of HSPB8 myopathy?
🦵 Muscle weakness (hips, shoulders)
💪 Muscle wasting
🚶 Difficulty walking
⚡ Cramps & fatigue
🐢 Slow progression over years
Each case is unique. Diagnosis often involves muscle biopsy & genetic testing. #HSPB8 #RareDisease #MyopathyAwareness #CureHSPB8
🦵 Muscle weakness (hips, shoulders)
💪 Muscle wasting
🚶 Difficulty walking
⚡ Cramps & fatigue
🐢 Slow progression over years
Each case is unique. Diagnosis often involves muscle biopsy & genetic testing. #HSPB8 #RareDisease #MyopathyAwareness #CureHSPB8
May 30, 2025 at 5:04 PM
What are the symptoms of HSPB8 myopathy?
🦵 Muscle weakness (hips, shoulders)
💪 Muscle wasting
🚶 Difficulty walking
⚡ Cramps & fatigue
🐢 Slow progression over years
Each case is unique. Diagnosis often involves muscle biopsy & genetic testing. #HSPB8 #RareDisease #MyopathyAwareness #CureHSPB8
🦵 Muscle weakness (hips, shoulders)
💪 Muscle wasting
🚶 Difficulty walking
⚡ Cramps & fatigue
🐢 Slow progression over years
Each case is unique. Diagnosis often involves muscle biopsy & genetic testing. #HSPB8 #RareDisease #MyopathyAwareness #CureHSPB8
🎧 New podcast alert!
Cure HSPB8 - The podcast just launched - and Ep. 1 is live!
We’re sharing our story, our mission, and our fight for a life free of HSPB8 Myopathy.
🧬 Listen here: rss.com/podcasts/cur...
#RareDisease #CureHSPB8 #HSPB8Myopathy #PatientAdvocacy #PodcastLaunch
Cure HSPB8 - The podcast just launched - and Ep. 1 is live!
We’re sharing our story, our mission, and our fight for a life free of HSPB8 Myopathy.
🧬 Listen here: rss.com/podcasts/cur...
#RareDisease #CureHSPB8 #HSPB8Myopathy #PatientAdvocacy #PodcastLaunch
May 23, 2025 at 5:02 PM
🎧 New podcast alert!
Cure HSPB8 - The podcast just launched - and Ep. 1 is live!
We’re sharing our story, our mission, and our fight for a life free of HSPB8 Myopathy.
🧬 Listen here: rss.com/podcasts/cur...
#RareDisease #CureHSPB8 #HSPB8Myopathy #PatientAdvocacy #PodcastLaunch
Cure HSPB8 - The podcast just launched - and Ep. 1 is live!
We’re sharing our story, our mission, and our fight for a life free of HSPB8 Myopathy.
🧬 Listen here: rss.com/podcasts/cur...
#RareDisease #CureHSPB8 #HSPB8Myopathy #PatientAdvocacy #PodcastLaunch
Our Program Director, Anna Kordala, is giving the Opening Keynote at #GenomicaConference in Cracow 🇵🇱 this week!
🎤 She’ll be diving into protein aggregation disorders & more. Connect if you’re attending!
🗓️ May 23–25
🎤 She’ll be diving into protein aggregation disorders & more. Connect if you’re attending!
🗓️ May 23–25
May 21, 2025 at 5:21 PM
Our Program Director, Anna Kordala, is giving the Opening Keynote at #GenomicaConference in Cracow 🇵🇱 this week!
🎤 She’ll be diving into protein aggregation disorders & more. Connect if you’re attending!
🗓️ May 23–25
🎤 She’ll be diving into protein aggregation disorders & more. Connect if you’re attending!
🗓️ May 23–25
🦄 CureHSPB8 is the only charitable initiative exclusively dedicated to addressing the challenges of #HSPB8Myopathy. Our mission starts with raising awareness and ensuring patients get the correct diagnosis, a key barrier for this rare condition.
Join us in making a difference! 💙 curehspb8.org
Join us in making a difference! 💙 curehspb8.org
May 16, 2025 at 5:50 PM
🦄 CureHSPB8 is the only charitable initiative exclusively dedicated to addressing the challenges of #HSPB8Myopathy. Our mission starts with raising awareness and ensuring patients get the correct diagnosis, a key barrier for this rare condition.
Join us in making a difference! 💙 curehspb8.org
Join us in making a difference! 💙 curehspb8.org
Living with a rare health condition like HSPB8 #myopathy can be challenging, but you are not alone. 💙 #CureHSPB8 is here to support patients, caregivers, and families through collaboration with @Orphanet, @to-cure-a-rose-foundation @CORDS @NORD @globalGenes @MDA OMIM , and more.
#RareDiseases
#RareDiseases
May 14, 2025 at 5:50 PM
Living with a rare health condition like HSPB8 #myopathy can be challenging, but you are not alone. 💙 #CureHSPB8 is here to support patients, caregivers, and families through collaboration with @Orphanet, @to-cure-a-rose-foundation @CORDS @NORD @globalGenes @MDA OMIM , and more.
#RareDiseases
#RareDiseases
Meet Sylwia Szwec, our new Research Program Manager at #CureHSPB8! With a PhD in progress on Duchenne Muscular Dystrophy and experience in drug regulation, Sylwia’s joining us to help turn science into therapies. #TeamScience #RareDisease
May 12, 2025 at 5:50 PM
Meet Sylwia Szwec, our new Research Program Manager at #CureHSPB8! With a PhD in progress on Duchenne Muscular Dystrophy and experience in drug regulation, Sylwia’s joining us to help turn science into therapies. #TeamScience #RareDisease
#HSPB8 = muscle guardian 💪
Prevents protein clumps
Recycles damaged proteins
Protects muscles during stress
A critical player in keeping your muscles healthy and functional!
#CureHSPB8 #RareDisease #PatientAdvocacy
Prevents protein clumps
Recycles damaged proteins
Protects muscles during stress
A critical player in keeping your muscles healthy and functional!
#CureHSPB8 #RareDisease #PatientAdvocacy
May 3, 2025 at 1:03 AM
#HSPB8 = muscle guardian 💪
Prevents protein clumps
Recycles damaged proteins
Protects muscles during stress
A critical player in keeping your muscles healthy and functional!
#CureHSPB8 #RareDisease #PatientAdvocacy
Prevents protein clumps
Recycles damaged proteins
Protects muscles during stress
A critical player in keeping your muscles healthy and functional!
#CureHSPB8 #RareDisease #PatientAdvocacy
We wrapped up an inspiring week at #WODC2025 in Boston! We are returning with renewed motivation and valuable connections to further our mission. Thank you to all who shared their knowledge and passion. #RareDisease #OrphanDrugs #CureHSPB8
April 29, 2025 at 4:04 PM
We wrapped up an inspiring week at #WODC2025 in Boston! We are returning with renewed motivation and valuable connections to further our mission. Thank you to all who shared their knowledge and passion. #RareDisease #OrphanDrugs #CureHSPB8
🚨 Big news: Cure HSPB8 has received new 2025 funding from @volofoundation.bsky.social!
This support helps us expand research, grow our patient registry, and drive awareness for #HSPB8Myopathy. Grateful for partners who believe in science & impact! 💙
#CureHSPB8 #RareDiseasen #PatientAdvocacy
This support helps us expand research, grow our patient registry, and drive awareness for #HSPB8Myopathy. Grateful for partners who believe in science & impact! 💙
#CureHSPB8 #RareDiseasen #PatientAdvocacy
April 26, 2025 at 1:02 AM
🚨 Big news: Cure HSPB8 has received new 2025 funding from @volofoundation.bsky.social!
This support helps us expand research, grow our patient registry, and drive awareness for #HSPB8Myopathy. Grateful for partners who believe in science & impact! 💙
#CureHSPB8 #RareDiseasen #PatientAdvocacy
This support helps us expand research, grow our patient registry, and drive awareness for #HSPB8Myopathy. Grateful for partners who believe in science & impact! 💙
#CureHSPB8 #RareDiseasen #PatientAdvocacy
🚀 Exciting news: We are heading to the #WorldOrphanDrugCongress USA 2025!
We’re on a mission to accelerate research for #HSPB8 #myopathy, a rare and progressive disease with no treatment — yet.
📍 Stop by our poster & let’s connect to make breakthroughs happen!
#RareDisease #HSPB8Myopathy
We’re on a mission to accelerate research for #HSPB8 #myopathy, a rare and progressive disease with no treatment — yet.
📍 Stop by our poster & let’s connect to make breakthroughs happen!
#RareDisease #HSPB8Myopathy
April 17, 2025 at 8:25 PM
🚀 Exciting news: We are heading to the #WorldOrphanDrugCongress USA 2025!
We’re on a mission to accelerate research for #HSPB8 #myopathy, a rare and progressive disease with no treatment — yet.
📍 Stop by our poster & let’s connect to make breakthroughs happen!
#RareDisease #HSPB8Myopathy
We’re on a mission to accelerate research for #HSPB8 #myopathy, a rare and progressive disease with no treatment — yet.
📍 Stop by our poster & let’s connect to make breakthroughs happen!
#RareDisease #HSPB8Myopathy
Our Program Director, Dr. Ania Kordala, is heading to Warsaw for the conference hosted by the European Economic and Social Committee (EESC)!
She’s representing our mission to accelerate research & support for #HSPB8 #myopathy — and all rare diseases.
www.eesc.europa.eu/en/agenda/ou... "
She’s representing our mission to accelerate research & support for #HSPB8 #myopathy — and all rare diseases.
www.eesc.europa.eu/en/agenda/ou... "
April 7, 2025 at 1:59 PM
Our Program Director, Dr. Ania Kordala, is heading to Warsaw for the conference hosted by the European Economic and Social Committee (EESC)!
She’s representing our mission to accelerate research & support for #HSPB8 #myopathy — and all rare diseases.
www.eesc.europa.eu/en/agenda/ou... "
She’s representing our mission to accelerate research & support for #HSPB8 #myopathy — and all rare diseases.
www.eesc.europa.eu/en/agenda/ou... "
🚨 Partnership Alert! 🚨
We’re teaming up with the To Cure A Rose Foundation to accelerate research and find a treatment for HSPB8-related neuromuscular diseases!
Collaboration = Faster progress. More hope. A future with cures.
#CureHSPB8 #RareDisease #ScienceForHope #CollaborationMatters
We’re teaming up with the To Cure A Rose Foundation to accelerate research and find a treatment for HSPB8-related neuromuscular diseases!
Collaboration = Faster progress. More hope. A future with cures.
#CureHSPB8 #RareDisease #ScienceForHope #CollaborationMatters
April 4, 2025 at 11:40 AM
🚨 Partnership Alert! 🚨
We’re teaming up with the To Cure A Rose Foundation to accelerate research and find a treatment for HSPB8-related neuromuscular diseases!
Collaboration = Faster progress. More hope. A future with cures.
#CureHSPB8 #RareDisease #ScienceForHope #CollaborationMatters
We’re teaming up with the To Cure A Rose Foundation to accelerate research and find a treatment for HSPB8-related neuromuscular diseases!
Collaboration = Faster progress. More hope. A future with cures.
#CureHSPB8 #RareDisease #ScienceForHope #CollaborationMatters
📢 The Cure HSPB8 2024 Annual Report is out!
This year, we made strides in research, advocacy & collaboration—all thanks to YOU. Check it out & see how we’re shaping the future of #RareDisease research.
🔗 Read here: static1.squarespace.com/static/654c0...
#CureHSPB8 #AnnualReport #ResearchMatters
This year, we made strides in research, advocacy & collaboration—all thanks to YOU. Check it out & see how we’re shaping the future of #RareDisease research.
🔗 Read here: static1.squarespace.com/static/654c0...
#CureHSPB8 #AnnualReport #ResearchMatters
March 24, 2025 at 10:53 PM
📢 The Cure HSPB8 2024 Annual Report is out!
This year, we made strides in research, advocacy & collaboration—all thanks to YOU. Check it out & see how we’re shaping the future of #RareDisease research.
🔗 Read here: static1.squarespace.com/static/654c0...
#CureHSPB8 #AnnualReport #ResearchMatters
This year, we made strides in research, advocacy & collaboration—all thanks to YOU. Check it out & see how we’re shaping the future of #RareDisease research.
🔗 Read here: static1.squarespace.com/static/654c0...
#CureHSPB8 #AnnualReport #ResearchMatters