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Today on bluesky I discuss the publication on Brogidirsen - the first in human clinical trial is now published in Cell Reports bsky.app/profile/oligog…

Read a very interesting paper today in Mary Ann Liebert Inc Human Gene Therapy by Laforet at al on TMA after AAV gene therapy. DOI: 10.1089/hum.2024.156 Full story on Bluesky 👇 bsky.app/profile/oligog…

Interested in my summary and comments on the nature paper by Means et al on the rapid method to generate IPSc and test personalized ASOs using Duchenne as a model disease? Read it on bluesky bsky.app/profile/oligog…

DMD & SMA Expert Masterclass – 8-10 July 2025, Prague TREAT-NMD™ invites healthcare professionals to a three-day, expert-led masterclass on Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) in Prague, Czech Republic. 🔹 Chaired by: Prof. Laurent Servais (MDUK

Share your experience of being a carrier and/or female carer of Duchenne muscular dystrophy (DMD) with researchers and at The John Walton Muscular Dystrophy Centre. To take part scan the QR code or follow the link redcap.link/myexperience

For my followers from Industry: there will be a TREAT-NMD masterclass for you! It will cover all you need to know about Duchenne (care, pathology, genetics, therapeutic options, considerations for preclinical and clinical research etc) tickettailor.com/events/treatnm…

Early morning to travel from -1 celsius to +30 at the first regional OTS Oligonucleotide Therapeutics Society meeting in Bengaluru. Looking forward to the meeting.

Greetings from Bangalore for the first Oligonucleotide Therapeutics Society Regional OTS meeting (starting tomorrow). My #apaperaday will focus on ASO work this week to commemorate. We kick off with a paper from Oligonucleotide Therapeutics Society Mary Ann Liebert Inc journal NAT and a senior author from India! bsky.app/profile/oligog…

🚀 TREAT-NMD’s DMD Industry Day Symposium – Secure Your Spot! Are you working in the pharmaceutical or biotech industry and looking to enhance your knowledge of Duchenne Muscular Dystrophy (DMD)? Join us in Prague on 11th July 2025 for a one-day expert-led symposium designed to

Join Our Mission to Advance Neuromuscular Care! TREAT-NMD Alliance Ltd. is now accepting applications for two key roles on our Board of Trustees: Chair and Trustee! These positions are crucial in guiding the strategic direction of TREAT-NMD, a charity dedicated to improving the

🚀 TREAT-NMD Duchenne Industry Day 💡✨ 📍 Prague | A key event for pharma & biotech professionals to explore: ✅ Outcome measures ✅ Genetic & treatment advancements ✅ International standards of care Engage, learn & connect with experts! 🔗tickettailor.com/events/treatnm… #TREATNMD

📅 Mark your calendar for the 21st Annual Oligo Meeting! This year's meeting will take place at the Budapest Congress Center in Budapest, Hungary, from October 19th- 22nd. 📅 Learn more at 2025oligomeeting.com #ots #oligotherapeutics #oligonucleotide #ots2025 #oligomeeting

If like me you never get enough of nucleic acid therapeutics conferences and realize that delivery is a challenge, the conference below might be of interest to you. #KSNATherapy26 keystonesymposia.org/conferences/co…

📅 SAVE THE DATE for the OTS Annual Meeting! Mark your calendar for the 21st Annual Oligo Meeting! It will be held at the Budapest Congress Center in Budapest, Hungary, from October 19th - 22nd. 📅 More information will be available soon. ⬇️ 2025oligomeeting.com #ots

Now available online - open access! Our review paper on the efforts of the N1 Collaborative to facilitate development of N=1 ASOs. N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing | Nucleic Acids Research | Oxford Academic

New paper out. Thanks to IRDiRC N=1 taskforce members for a very positive, proactive and pleasant collaboration N1 Collaborative n-Lorem Foundation Tim Yu and others! bsky.app/profile/oligog…

A new paper out in Neuromuscular Disorders from Alper Yavas, who recently obtained his PhD on using antisense oligonucleotides to redirect polyadenylation sites in the IGF1 transcripts (very complex gene with alternative splicing and adenylation) sciencedirect.com/science/articl…

Collaborative paper out! This work started at #OTSLeiden Oligonucleotide Therapeutics Society in 2015 where 2 groups from LUMC discovered a mutual interest in splicing. Indeed we met at the OTS meeting for the first time and not at LUMC liebertpub.com/doi/10.1089/na…

Masad Damha’s journey from a curious student in Nicaragua to a pioneering chemist in the field of RNA research illustrates the profound impact of education, collaboration, and mentorship in scientific discovery. oligotherapeutics.org/ots-member-hig… #ots25 #oligomeeting #oligotherapeutics

Are you HIRING or LOOKING for a position in Oligo Therapeutics? The OTS Job Board makes the process easy for both employers and candidates. 👉 Get started today at oligotherapeutics.org/job-postings/ #oligotherapeutics #oligonucleotide #OTS #jobs #jobboard #science #biotechnology