Journal of Neuromuscular Diseases
@journalnd.bsky.social
Facilitates progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic NDMs. Editors: Carsten Bönnemann and Hanns Lochmüller. https://journals.sagepub.com/home/JND
🫀 Myocarditis is a serious risk after gene therapy for Duchenne muscular dystrophy (DMD). In this new review paper, Kaufman et al. call for proactive cardiac monitoring & risk stratification to safeguard patients receiving AAV-based therapies. #DMD #GeneTherapy #Myocarditis
🔗 buff.ly/mhlJVGK
🔗 buff.ly/mhlJVGK
May 16, 2025 at 11:03 AM
🫀 Myocarditis is a serious risk after gene therapy for Duchenne muscular dystrophy (DMD). In this new review paper, Kaufman et al. call for proactive cardiac monitoring & risk stratification to safeguard patients receiving AAV-based therapies. #DMD #GeneTherapy #Myocarditis
🔗 buff.ly/mhlJVGK
🔗 buff.ly/mhlJVGK
Knowledge on early motor milestones in #DMD is limited, but this is essential given increasing newborn screening and earlier age groups in clinical trials.
📑 This research study by Lowes et al sheds new light on DMD in <5 year age group.
➡️ Full report here buff.ly/x1Y7sCl
📑 This research study by Lowes et al sheds new light on DMD in <5 year age group.
➡️ Full report here buff.ly/x1Y7sCl
May 15, 2025 at 4:07 PM
Knowledge on early motor milestones in #DMD is limited, but this is essential given increasing newborn screening and earlier age groups in clinical trials.
📑 This research study by Lowes et al sheds new light on DMD in <5 year age group.
➡️ Full report here buff.ly/x1Y7sCl
📑 This research study by Lowes et al sheds new light on DMD in <5 year age group.
➡️ Full report here buff.ly/x1Y7sCl
From our latest issue 🆕 research from @uottawa.bsky.social demonstrating peripheral effects before motor neuron loss in the #SMA 2B/- mouse model. Read the #openaccess paper here: buff.ly/51H530o #spinalmuscularatrophy #neuromuscularjunction
May 12, 2025 at 4:09 PM
From our latest issue 🆕 research from @uottawa.bsky.social demonstrating peripheral effects before motor neuron loss in the #SMA 2B/- mouse model. Read the #openaccess paper here: buff.ly/51H530o #spinalmuscularatrophy #neuromuscularjunction
New insights into LAMA2 related dystrophies using international registry data @cureCMD - essential info on early clinical findings and natural history. Check out the full #openaccess paper here buff.ly/1zO5RUn
May 8, 2025 at 11:03 AM
New insights into LAMA2 related dystrophies using international registry data @cureCMD - essential info on early clinical findings and natural history. Check out the full #openaccess paper here buff.ly/1zO5RUn
New original research from Manon et al provides novel insights into #myotonicdystrophy type 2, from the Dutch neuromuscular database. Their study shows ncreased risk of cardiac disease, pneumonia, and malignancies. Read the full report here: buff.ly/LhquCrS
May 5, 2025 at 11:02 AM
New original research from Manon et al provides novel insights into #myotonicdystrophy type 2, from the Dutch neuromuscular database. Their study shows ncreased risk of cardiac disease, pneumonia, and malignancies. Read the full report here: buff.ly/LhquCrS
NEW research paper in JND = open label extension study of the neonatal Fc receptor inhibitor Rozanolixizumab #MyastheniaGravis:
- Clinically meaningful and consistent improvement
- Acceptable safety profile
- Beneficial in AChR and MuSK MG
Read the report here journals.sagepub.com/doi/full/10....
- Clinically meaningful and consistent improvement
- Acceptable safety profile
- Beneficial in AChR and MuSK MG
Read the report here journals.sagepub.com/doi/full/10....
April 30, 2025 at 7:49 AM
NEW research paper in JND = open label extension study of the neonatal Fc receptor inhibitor Rozanolixizumab #MyastheniaGravis:
- Clinically meaningful and consistent improvement
- Acceptable safety profile
- Beneficial in AChR and MuSK MG
Read the report here journals.sagepub.com/doi/full/10....
- Clinically meaningful and consistent improvement
- Acceptable safety profile
- Beneficial in AChR and MuSK MG
Read the report here journals.sagepub.com/doi/full/10....
How many countries are screening newborns for #SMA? A new paper by Vrščaj et al surveys 86 experts from 80 countries on current implementation, therapy access, and future projections. Read the full paper here: https://journals.sagepub.com/doi/full/10.1177/22143602241288095
February 17, 2025 at 4:47 PM
How many countries are screening newborns for #SMA? A new paper by Vrščaj et al surveys 86 experts from 80 countries on current implementation, therapy access, and future projections. Read the full paper here: https://journals.sagepub.com/doi/full/10.1177/22143602241288095
The latest issue of JND can be found here: journals.sagepub.com/toc/JND/curr...
Here you can also find the author and submission guidelines, editorial board and further journal statistics.
Here you can also find the author and submission guidelines, editorial board and further journal statistics.
February 10, 2025 at 10:07 AM
The latest issue of JND can be found here: journals.sagepub.com/toc/JND/curr...
Here you can also find the author and submission guidelines, editorial board and further journal statistics.
Here you can also find the author and submission guidelines, editorial board and further journal statistics.