#myopathies
A small study found promising results using CAR T-cell therapy to treat autoimmune diseases by targeting Epstein-Barr virus (EBV)-infected immune cells.

• CAR T-cell therapy: 8 of 12 achieved drug-free remission
• Rituximab: 0 of 15 achieved remission

Promising, but more research is needed.
November 3, 2026 at 4:48 AM
Drug-induced myopathies are a common cause of muscle pain, and the range of culprit drugs is constantly expanding. This review covers the diagnostic and therapeutic process and the spectrum of drug-induced myopathies 💊💪 #Myopathy #DrugSafety
mdpi.com/2227-9059/12...
November 4, 2026 at 12:54 AM
📄New from the #Transfusion Evidence Alert:
Intravenous Immunoglobulin Add-On in Newly Diagnosed Idiopathic Inflammatory Myopathies: A Randomized Clinical Trial
By Özkaynar et al.

🔗Transfusion Evidence Library link in replies
#MedSky
October 30, 2026 at 9:26 AM
Transfusion Evidence Library link to:
Intravenous Immunoglobulin Add-On in Newly Diagnosed Idiopathic Inflammatory Myopathies: A Randomized Clinical Trial by Özkaynar et al.
www.transfusionevidencelibrary.com/alerts/artic...
Intravenous Immunoglobulin Add-On in Newly Diagnosed Idiopathic Inflammatory Myopathies: A Randomized Clinical Trial
IMPORTANCE: Preliminary evidence suggests that intravenous immune globulin (IVIG) may be valuable as an add-on treatment in newly diagnosed idiopathic inflammatory myopathies (IIMs), but a randomized ...
www.transfusionevidencelibrary.com
October 30, 2026 at 9:27 AM
In accordance with Françoise’s final wishes, donations in her memory will go to AFM-Téléthon to support the fight against myopathies and neuromuscular diseases. 🖤

mapage.telethon.fr/collecte-hom...
En mémoire de Françoise Helmbacher
Soutenez le Téléthon en faisant un don sur la page de En mémoire de Françoise Helmbacher via https://mapage.telethon.fr/collecte-hommages/En-memoire-de-Francoise-Helmbacher
mapage.telethon.fr
October 26, 2026 at 8:25 AM
This review summarizes current and emerging therapies for adult idiopathic inflammatory myopathies, with an emphasis on their clinical relevance, phenotype-specific application, and the evolving role of biomarker-informed #PrecisionMedicine in #refractorydisease.
doi.org/10.59717/j.x...
October 27, 2026 at 2:50 AM
Pierre Birambeau, co-fondateur du Téléthon et son directeur de 1987 à 2002, est décédé à l'âge de 84 ans, annonce le conseil d'administration de l'AFM-Téléthon #téléthon #PierreBirambeau #décès
Pierre Birambeau, co-fondateur du Téléthon et son directeur de 1987 à 2002, est décédé à l'âge de 84 ans, annonce le conseil d'administration de l'AFM-Téléthon
Pierre Birambeau, l’un des fondateurs du Téléthon français, est décédé hier à l’âge de 84 ans, a annoncé ce jeudi l’Association française contre les myopathies (AFM-Téléthon). Sa...
www.jeanmarcmorandini.com
October 29, 2026 at 12:28 PM
Pierre Birambeau, co-fondateur du Téléthon et son directeur de 1987 à 2002, est décédé à l'âge de 84 ans, annonce le conseil d'administration de l'AFM-Téléthon #téléthon #PierreBirambeau #décès
Pierre Birambeau, co-fondateur du Téléthon et son directeur de 1987 à 2002, est décédé à l'âge de 84 ans, annonce le conseil d'administration de l'AFM-Téléthon
Pierre Birambeau, l’un des fondateurs du Téléthon français, est décédé hier à l’âge de 84 ans, a annoncé ce jeudi l’Association française contre les myopathies (AFM-Téléthon). Sa...
www.jeanmarcmorandini.com
October 29, 2026 at 12:28 PM
Expansion of Newborn Screening for Rare Myopathies - read more
October 23, 2026 at 3:10 AM
Pomi Yun, Lourdes Figueroa, Chang Seok Lee, Filip Van Petegem, Nicol C Voermans, Robert T Dirksen, Michael F Goldberg, Līvija Medne, Jinson Paul, Mairéad Ryan, Emily Pedersen, Johanna Lanner, and Payam Mohassel
<em>RYR1</em>-related diseases patient-led international research workshop: Novel perspectives, treatments and interventions
RYR1-related diseases (RYR1-RDs) encompass a wide spectrum of disease manifestations with limited available disease-modifying therapies. The RYR1-RD phenotypes include congenital myopathies of different histotypes often with early-onset muscle weakness, feeding difficulties, and respiratory insufficiency of varying severity. RYR1-RDs also include a group of episodic diseases such as exercise intolerance, exertional rhabdomyolysis, periodic paralysis, and malignant hyperthermia susceptibility....
journals.sagepub.com
October 23, 2026 at 4:00 AM
New Podcast Episode Available! 🎙️

Episode 14 | Journal Club: Iannibelli, Ruggieri et al., 2025
Aggrephagy markers elevated across all MFM subtypes - a possible shared disease mechanism. We discuss what this means for understanding and treating MFMs.
buff.ly/hTNBD05
EP 14: Journal Club: Iannibelli, Ruggieri et al., 2025 | Aggrephagy Markers in Myofibrillar Myopathies | Cure MFM13 - The Podcast
In this Journal Club episode, we discuss the publication by Iannibelli, Ruggieri and colleagues investigating the role of aggrephagy - the cellular…
buff.ly
October 19, 2026 at 5:04 PM
Asthma medication improved motor skills and strength in children with muscle disease
A common asthma medication improves motor skills and muscle strength in children with congenital myopathies, a group of incurable muscle diseases. In a study, c...
weiterlesen
October 14, 2026 at 4:49 AM
An asthma drug improved motor function in a small congenital myopathy trial. The 11 children able to walk covered 38 more meters in six minutes during treatment; larger trials are needed. doi.org/hcks4w
Asthma drug improves motor function and strength in children with rare muscle diseases in small trial
Congenital myopathies are a group of rare, inherited muscle diseases that often cause muscle weakness in childhood. This weakness can affect, among other things, the ability to walk, stand up and use one's arms and hands, and in some cases, it can also affect the respiratory muscles.
medicalxpress.com
October 14, 2026 at 4:20 PM
6/6 👏 Bravo au Dr Svetlana Gorokhova, aux équipes AP-HM de génétique médicale et de PACARARE, et aux contributeurs FILNEMUS !

L’article PEMR-APHM 👇
Myopathies : mise à jour 2025 des listes de gènes pour le diagnostic | AP-HM
Le diagnostic des myopathies peut être complexe en raison de leur grande diversité génétique. Une étude coordonnée par le Dr Svetlana GOROKHOVA, sous l’égide de la Commission Génétique moléculaire de la Filière Nationale des Maladies Rares Neuromusculaires FILNEMUS (coordination Pr Mire...
fr.ap-hm.fr
October 13, 2026 at 7:04 AM
1/6 #MardiActu #MaladiesRares 🧬

Myopathies : mieux cibler les analyses génétiques pour faire avancer le diagnostic.

La mise à jour 2025 des listes de gènes, portée par FILNEMUS, vise à harmoniser les pratiques et à améliorer le parcours des patients. On vous explique 👇
October 13, 2026 at 7:04 AM
Extract: The position involves experimental research on skeletal muscle dysfunction in inflammatory myopathies, requiring a PhD in medical science or related fields and fluency in Swedish.
October 5, 2026 at 2:06 PM
An interesting look back at 50 years of juvenile myositis research and progress. It’s remarkable to see how far our understanding of these rare diseases has come — and how much has been built through decades of collaboration and dedication. 💙

pmc.ncbi.nlm.nih.gov/articles/PMC...
Juvenile Myositis: Insights on Classification, Pathogenesis and Therapies over the Past 50 Years
The juvenile idiopathic inflammatory myopathies (JIIM) are rare serious, multisystem autoimmune diseases affecting children and young people. Fifty years ago the prognosis for these conditions was poo...
pmc.ncbi.nlm.nih.gov
September 30, 2026 at 12:32 PM
NEW RESEARCH—Minimal residual disease flow cytometry of peripheral blood B-cell subsets in patients with idiopathic inflammatory myopathies following rituximab therapy: a single-centre, retrospective cohort study thelancet.com/journals/lan...
#MedSky #RheumSky
September 25, 2026 at 4:28 PM
NEW RESEARCH—Minimal residual disease flow cytometry of peripheral blood B-cell subsets in patients with idiopathic inflammatory myopathies following rituximab therapy: a single-centre, retrospective cohort study www.thelancet.com/journals/lan... #MedSky #RheumSky
September 25, 2026 at 10:36 AM
Very interesting how we describe that cell cortex in myofibers is involved in tubules formation in muscle cells, with implication for centronuclear myopathies and other triadopathies. Happy to lead this work with @raquel-pereira.bsky.social @drmichaelway.bsky.social and other co authors.
September 20, 2026 at 1:20 PM