En unos días la colgaran en YouTube y la paso...

Presentación de @varechavala.bsky.social sobre los Ensayos Clįnicos, los Registros de Pacientes, las Asociaciones de Pacientes de #EnfermedadesRaras en #NaukasBilbao2025 (desde el minuto 15) www.eitb.eus/es/nahieran/...

#naukasbilbao25 Virginia Arechavala Gomeza @varechavala.bsky.social @[email protected] nos cuenta que si tú participas en un ensayo puedes dificultar que un medicamento llegue antes al mercado, pues tú puedes ser el unicornio que dificulta el análisis del ensayo (los criterios de inclusión)

Virginia @varechavala.bsky.social #Ikerbasque nos ha dado una charla magnífica en @naukas.bsky.social #NaukasBilbao2025 sobre los ensayos clínicos, sobre su importancia y sobre su objetivo fundamental: poder decidir si esa terapia propuesta además de segura es eficaz, si funciona o no. Enhorabuena!

«No es ni una cura ni un último tren. Es un experimento» Virginia Arechavala Gomeza Entendiendo que criterios hay para entrar o no en un ensayo clínico. #NaukasBilbao2025 #NaukasBilbao15aniversario #NaukasBilbao

12/ 🧬 También nos explica sus avances con #CaixaImpulse Innovación Beatriz Llamusí, directora científica y cofundadora de Arthex Biotech, que ha desarrollado una terapia de ARN para la distrofia miotónica. 🗣️👇

German study: Efficacy and safety of respiratory strength and endurance training in patients with myotonic dystrophy type 1 (DM1): a randomized controlled trial link.springer.com/article/10.1...

Today we're supporting international myotonic dystrophy awareness day! #MyotonicDystrophy (DM) is an inherited multisystem condition that mainly causes progressive muscle loss, weakness and myotonia. Join us in raising awareness and supporting research: bit.ly/41Wng82

AMO Pharma Unveils Promising Long-Term Safety Data for AMO-02 in Congenital Myotonic Dystrophy Type 1 Treatment#USA#London#Myotonic_Dystrophy#AMO_Pharma#AMO-02

Today is International #MyotonicDystrophy Awareness Day. Myotonic dystrophy may affect as many as 1 in 2,100 people across the world. Our tower will be illuminated in green this week to show support on the 5th annual awareness day 💚 More ➡️ myotonic.org

Monday night, landmarks across New York State were lit green to mark International Myotonic Dystrophy Awareness Day. Scientists at #UAlbany's RNA Institute are leaders in #MyotonicDystrophy research & were behind New York's green glow this week. cbs6albany.com/news/local/l...

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1): protocol of an international natural history study https://www.medrxiv.org/content/10.1101/2025.08.13.25333647v1

The CMR gathers wearing green to celebrate International Myotonic Dystrophy Awareness Day to promote research & funding to improve the lives of those affected by DM, a neuromuscular disease causing progressive muscle weakness @mda.org @myotonicstrong.bsky.social #muscle

Japanese study: Validation of the FVB/N-Tg(HSA* LR)20bCath mice of myotonic dystrophy using swallowing function assessment, histology, and immunofluorescence analysis journals.plos.org/plosone/arti...

Univ of Minnesota/Tufts study: The impact of Hnrnpl deficiency on transcriptional patterns of developing muscle cells febs.onlinelibrary.wiley.com/doi/10.1002/...

🌍 60+ Global Alliance Organizations Recognize the 5th annual International #MyotonicDystrophy Awareness Day! 💚 We proudly celebrate the remarkable strength & resilience of the DM community around the world. 🌟 Read the full press release at www.myotonic.org/global-allia...

People living with #myotonicDystrophy have a 50% chance of passing it on to their children. Early genetic testing and counseling can save lives! Help change the future of #myotonicDystrophy on September 15! Learn more at: www.myotonic.org/internationa...

There's still time to Go #GreenForDM! Raising #myotonicDystrophy awareness is as simple as wearing green 💚 Join the MDF Team and the DM community — post your pic, use #myotonicDystrophy, and tag MDF to show your support on Awareness Day! 💪✨

¡Ahora disponible en español – Infografía sobre el ejercicio con #distrofiaMiotónica! El ejercicio puede optimizar la función corporal y mantener la fuerza. #myotonicDystrophy 👉 www.myotonic.org/sites/defaul...

¡Ahora en español: Recomendaciones sobre anestesia y #distrofiaMiotónica! La anestesia puede ser riesgosa en DM; este recurso ofrece información crucial para pacientes y familias. #myotonicDystrophy 👉 www.myotonic.org/sites/defaul...

We support International #MyotonicDystrophy Awareness Day! 💚 Learn more about research & trials conducted by our teams⤵️ #myotonicDystrophy #myotonicDystrophyAwareness @afm-telethon.bsky.social @afmtelethonpresse.bsky.social @myotonicstrong.bsky.social www.institut-myologie.org/2025/09/15/j...

Wearing our greens in the lab at Biobizkaia for #MyotonicDystrophy awareness day. @myotonicstrong.bsky.social

ARTHEx Biotech Upsizes Series B Financing Round to $87M to Advance Lead Program ATX-01 in Myotonic Dystrophy Type 1 and Expand Pipeline of Targeted RNA Medicines www.prnewswire.com/news-release...

Polish study: Promoter-targeted small RNA duplexes increase MBNL1 transcription and mitigate myotonic dystrophy-associated spliceopathy academic.oup.com/nar/article/...

Danish/Norwegian study: Living and coping with adult-onset myotonic dystrophy type 1 from the perspectives of caregivers www.researchgate.net/publication/...

Japanese study: Three cases of pregnancies complicated with myotonic dystrophy type 1 obgyn.onlinelibrary.wiley.com/doi/10.1111/...

Hungarian study: Measuring Myotonia: Normative Values and Comparison with Myotonic Dystrophy Type 1 www.mdpi.com/2035-8377/17...

Italian study: circARHGAP10 as a candidate biomarker and therapeutic target in myotonic dystrophy type 1 www.sciencedirect.com/science/arti...

Sanofi publication: Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1 www.sciencedirect.com/science/arti...

New #MolTherapy paper: Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1. #Myoblue tinyurl.com/mrjrue3f

We are joining hands with @myotonicstrong.bsky.social to raise awareness of myotonic dystrophy this month. Did you know that September 15 is the International Myotonic Dystrophy Awareness Day? #myotonicDystrophy #myotonicDystrophyAwareness

Myotonic dystrophy (DM) may affect as many as 1 in 2,100 individuals across the world. Make it known in honor of International #myotonicDystrophy Awareness Day on Sept 15! Learn more at: www.myotonic.org/internationa...

#myotonicDystrophy is commonly referred to as DM, an abbreviation of the Latin dystrophia myotonica. DM's other names include myotonic muscular dystrophy (MMD), Steinert’s Disease for DM1, and proximal myotonic myopathy (PROMM) for DM2. Learn more at: www.myotonic.org/internationa...

Myotonic dystrophy is the most common form of adult muscular dystrophy and is considered the most variable of all known conditions. Help make it the most well-known on International #myotonicDystrophy Awareness Day, September 15! Learn more at: www.myotonic.org/internationa...

Chinese study: Muscle-driven spinal cord histological and transcriptomic alterations in a myotonic dystrophy mouse model: insights into neuropathy academic.oup.com/braincomms/a...

Myotonic dystrophy is the most common form of adult muscular dystrophy. Join us to spread awareness about #myotonicDystrophy on September 15!

Myotonic dystrophy symptoms usually become more severe with each generation, yet there is currently no cure and no approved treatments. Help us get closer to better care and a cure by spreading #myotonicDystrophy awareness on September 15! Learn more at: www.myotonic.org/internationa...

Proud to join the Myotonic Dystrophy Foundation as a Global Alliance member! We'll help raise awareness on International #myotonicDystrophyAwareness Day, on Monday 9/15. Our lead program: an RNA-targeted small molecule drug for the treatment of myotonic dystrophy type 1 (DM1). #myotonicDystrophy

In honor of International Myotonic Dystrophy Awareness Day (Sept15), these buildings will be lighting up in Green. If you are near any of these buildings and happen to see them, please share a photo with us and/or share with us here and tag us. @myotonicstrong.bsky.social #myotonicdystrophyawareness

The Teesside youngster was diagnosed with a rare condition, Congenital Myotonic Dystrophy, as a baby

Bottlebrush Polymer Conjugates for Enhanced Antisense Oligonucleotide Therapy in Myotonic Dystrophy Type 1 https://www.biorxiv.org/content/10.1101/2025.08.06.668589v1

Living with myotonic dystrophy type 1 means carrying a weight most can’t see. ✏️Sokhna MS Yakhine-Diop, on how greater awareness, research, and compassion can lighten the load: daily27.info/2025/08/11/l... #MyotonicDystrophy #RareDisease #Neurodegenerative #News

New #hmg_journal paper: Transcriptome alterations underlying metabolic dysfunction and liver disease in myotonic dystrophy type 1. #Myoblue tinyurl.com/49m4w24c

MBNL Depletion Induces Muscle Stem Cell Activation and Myofibre Fusion while Impairing Myonuclear Maturation in Myotonic Dystrophy Type 1 #SingleCell 🧪🧬🖥️ https://www.researchsquare.com/article/rs-7311936/latest

Patients with myotonic dystrophy exhibited higher proportions of endocrine, gynecologic, and hematologic cancers but lower respiratory #cancer compared to the general population. ja.ma/3JumnwN

Univ. of Florida study: Differential pathology and susceptibility to MBNL loss across muscles in myotonic dystrophy mouse models insight.jci.org/articles/vie...

Univ. of Albany/VCU/$DYN study: Changes in RNA splicing as a surrogate endpoint for myotonic dystrophy Type 1 (DM1) clinical trials journals.sagepub.com/doi/10.1177/...

Dutch review: The blind men and the elephant: recognising the multisystem symptoms of myotonic dystrophy type 1 ojrd.biomedcentral.com/articles/10....

NIH/NCI/Yale/Stanford study: Spectrum of Cancers and Their Prognosis Among Patients With Myotonic Dystrophy jamanetwork.com/journals/jam...