#nusinersen
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blindendachs.bsky.social
- Die RSV-Impfung
- Nusinersen und Gentherapie bei SMA
- Gentherapie bei Zystischer Fibrose
February 12, 2026 at 6:29 PM
shihcheng.bsky.social
High-dose nusinersen (50mg loading, 28mg maintenance) in SMA phase 2/3 trial shows promise; potential for better outcomes. PMID:41634391, Nat Med 2026, @NatureMedicine https://doi.org/10.1038/s41591-025-04193-6 #Medsky #Pharmsky #RNA #ASHG #ESHG 🧪
High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial | Nature Medicine
Despite the remarkable benefits of nusinersen and other disease-modifying therapies in spinal muscular atrophy (SMA), patients may still experience clinical manifestations of the disease. Here we assessed the potential for high-dose nusinersen to rapidly slow neurodegeneration and lead to improved outcomes for patients. The global, three-part, phase 2/3 DEVOTE trial evaluated the efficacy and safety of high-dose nusinersen (50-mg loading dose; 28-mg maintenance dose) in individuals with SMA. In Part B, treatment-naive individuals (n = 75) were randomized 2:1 to 50/28 mg or 12/12 mg nusinersen. In a supportive open-label cohort (Part C), nusinersen-experienced individuals (12/12 mg for more than 1 year) were enrolled. The primary endpoint (Part B infantile-onset participants) was a 6-month change in the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score comparing 50/28 mg with matched ENDEAR participants (n = 20) who received sham. DEVOT
doi.org
March 4, 2026 at 2:10 AM
natrevneurol.nature.com
High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial www.nature.com/articles/s41...
High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial - Nature Medicine
The phase 2/3 DEVOTE trial demonstrated that high-dose nusinersen significantly improved motor function and was safe in patients with spinal muscular atrophy, compared with a matched sham control.
www.nature.com
February 3, 2026 at 12:55 PM
neurologieessen.bsky.social
Neue Publikation:
Neuronales Pentraxin 2 als potenzieller Biomarker für das Ansprechen auf die Nusinersen-Therapie bei Erwachsenen mit spinaler Muskelatrophie: Eine Pilotstudie

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August 4, 2025 at 2:03 PM
oligogirl.bsky.social
Publication out: Together with Prof Shin'ichi Takeda I wrote a historical perspective on eteplirsen and nusinersen: knowing where we are now, can we look back and see whether this could have been predicted, to also learn for future developments?
journals.sagepub.com/doi/10.1177/...
A historical perspective on the development of antisense oligonucleotide treatments for Duchenne muscular dystrophy and spinal muscular atrophy - Annemieke Aartsma-Rus, Shin'ichi Takeda, 2025
Splice modulating antisense oligonucleotides (ASOs) have been approved for the treatment of spinal muscular atrophy (nusinersen) and Duchenne muscular dystrophy...
journals.sagepub.com
March 5, 2025 at 9:54 AM
dzne.science
New paper out: Compound Muscle Action Potential (CMAP) Amplitude Trajectories and Pattern in Adults with 5q-Spinal Muscular Atrophy Receiving Nusinersen Therapy: A Multicenter, Binational Observational Study.: Bjelica, Bogdan; Wohnrade, Camilla; Osmanovic, Alma; et… Thanks for sharing! #dzne #papers
Compound Muscle Action Potential (CMAP) Amplitude Trajectories and Pattern in Adults with 5q-Spinal Muscular Atrophy Receiving Nusinersen Therapy: A Multicenter, Binational Observational Study.
Bjelica, Bogdan; Wohnrade, Camilla; Osmanovic, Alma; et al. European journal of neurology Vol. 32, no. 11, p. e70405
dlvr.it
November 19, 2025 at 12:26 PM
neurologieessen.bsky.social
Vergleichsstudie der motorischen Funktion bei Erwachsenen mit spinaler Muskelatrophie unter Nusinersen vs. Risdiplam
💉📅🚶‍➡️👨‍🦽‍➡️🏃‍♀️‍➡️💪🆚🥛📅🚶‍➡️👨‍🦽‍➡️🏃‍♀️‍➡️💪🟰🚶‍➡️👨‍🦽‍➡️🏃‍♀️‍➡️💪✅

1/4
June 1, 2026 at 2:53 PM
europeanjournalofneurology.ean.org
Quantitative MRI detected disease progression with higher sensitivity than conventional clinical outcome measures in nusinersen-treated adults with spinal muscular atrophy, supporting its use as an objective monitoring tool.

Read now: onlinelibrary.wiley.com/doi/10.1111/...

@ean.org #WileyNeuro
April 16, 2026 at 3:05 PM
oligogirl.bsky.social
Not discussed is the difference between nusinersen & tofersen, where nusinersen seems better tolerated. We can speculate: nusinersen is fully modified while tofersen has a gap for RNase H activation. We know from systematic ASOs that MOEs protect against the inflammatory response ASOs can induce.
March 19, 2025 at 11:26 AM
europeanjournalofneurology.ean.org
Coratti et al. demonstrate a persistent impact of nusinersen on clinical progression, most pronounced in younger patients with higher baseline HFMSE scores.

📚 Read the trending article now: buff.ly/s1jNEwz

#Neurology #SMA

@ean.org #WileyNeuro
August 8, 2025 at 4:02 PM
journalnd.bsky.social
📄 Next from the JND special issue on RNA therapeutics:
A focused review of real-world evidence for nusinersen in #SMA.

Across thousands of treated patients worldwide, data supports sustained motor benefits and a favourable safety profile.
Read it here: buff.ly/WISRWey

#Neuromuscular #RareDisease
Sage Journals: Discover world-class research
Subscription and open access journals from Sage, the world's leading independent academic publisher.
buff.ly
March 12, 2026 at 8:49 AM
world-j-pediatr.bsky.social
Nusinersen treatment is not only effective but also sustainable in improving and maintaining the motor function of Chinese children with later-onset spinal muscular atrophy.

#SoMe4PedSurg #MedEd #MedX #Pediatrics #Efficacy #Nusinersen #SMA #MotorFunction #TICMED25

link.springer.com/article/10.1...
August 1, 2025 at 9:19 AM
rarediseaseadvisor.bsky.social
A high-dose #Nusinersen regimen significantly improved motor function and reduced a biomarker of #Neurodegeneration in infants with spinal muscular atrophy (#SMA). Findings in @natmed.nature.com

Read more: https://bit.ly/4aFcY0A

#RareDisease #MedSky #SpinalMuscularAtrophy
High-Dose Nusinersen Offers Greater Benefits While Maintaining Safety in SMA
A high-dose nusinersen regimen may offer greater benefit in SMA than the standard regimen while maintaining comparable safety.
bit.ly
February 9, 2026 at 4:46 PM
mattmuscleguy.bsky.social
New #jclinicalinvest paper: Open-label phase 4 trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. #Myoblue tinyurl.com/5mybknx6
JCI - Open-label phase 4 trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy
tinyurl.com
September 23, 2025 at 7:29 PM
aycasahin.bsky.social
Today marks the 22nd dose of Spinraza (nusinersen). Exactly 10 days ago, the EMA approved the high-dose of nusinersen. We are now hoping—and urging—our country to follow the EMA’s decision and approve and reimburse high-dose Spinraza as soon as possible.
January 22, 2026 at 8:09 PM
bild.bsky.social
Clinical relevance of zebrafish for gene variants testing. Proof-of-principle with SMN1/SMA - EMBO Molecular Medicine
Spinal muscular atrophy (SMA) results from SMN1 gene loss-of-function (LOF), with disease severity directly linked to the level of remaining SMN protein. Nusinersen, risdiplam, and onasemnogene abepar...
link.springer.com
January 28, 2026 at 2:27 PM
neurologylive.bsky.social
🧬 Real-world data suggest motor improvements in children with #SMA after switching to #genetherapy.

A Polish study found patients transitioning from nusinersen or risdiplam to onasemnogene abeparvovec showed gains on motor scales within 6 months. Read more on NeurologyLive!

#Neurology #RareDisease
March 9, 2026 at 1:13 PM
theakarifoundation.bsky.social
El estudio DEVOTE fue diseñado para evaluar la dosis más elevada de nusinersen en todo el rango de edad, niveles de severidad de la enfermedad y estatus funcional. 🧬

🔗Lee el artículo completo en el siguiente link: lnkd.in/eS2FHNyc

#TheAkariFoundation #AtrofiaMuscularEspinal #Biogen #nusinersen
August 28, 2025 at 4:06 PM
oligogirl.bsky.social
The discussion sadly is mainly a rehash of the results rather than discussing 'what does this mean for nusinersen treatment?'. Authors briefly mention side effects of nusinersen, such as increased temperature, vomiting and constipation, but some of these are due to the procedure (lumbar punction).
February 4, 2026 at 7:45 AM
aycasahin.bsky.social
Today is my 23rd dose of Spinraza (nusinersen)! 🥰

If I were in Germany or the US, I would likely be receiving the first dose of high-dose regimen (50 mg/5 mL). In my country, it is still not approved, and we are still waiting. We need high-dose Spinraza in Türkiye!
May 14, 2026 at 8:14 PM
neurologieessen.bsky.social
Hier der Link zur englischen Veröffentlichung:
link.springer.com/article/10.1... via @springer.springernature.com

@unidue.bsky.social

4/4
Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam - Journal of Neurology
Background Nusinersen and risdiplam are approved disease-modifying therapies for adults with 5q-associated spinal muscular atrophy (SMA). To date, no direct comparison of the two treatments in adults ...
link.springer.com
June 1, 2026 at 2:53 PM
oligogirl.bsky.social
#apaperaday Forced to work from home due to a lack of trains (to Yuzu's delight). The pick is from @narjournal.bsky.social molecular insights by Ottesen et al on a study of off target effects of a MOE/PS ASO (the title does not mention the name, but it is nusinersen). doi: 10.1093/narmme/ugag002
February 4, 2026 at 7:18 AM
neurologieessen.bsky.social
Der NfL-Spiegel sank während der Nusinersen-Behandlung, was auf ein Potenzial als pharmakodynamischer Wirksamkeitsmarker bei SMA-Patienten hindeutet.

2/3
June 27, 2025 at 2:36 PM
neurologieessen.bsky.social
Neue Publikation:
Langzeitdynamik der Neurofilament-Leichtkette in Liquor und Serum bei erwachsenen Patienten mit 5q-spinaler Muskelatrophie unter Nusinersen-Behandlung

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June 27, 2025 at 2:36 PM
neurologylive.bsky.social
#Breaking: 🚨 The @fda.gov has approved a higher-dose regimen of nusinersen (Spinraza; Biogen) for Spinal Muscular Atrophy.

The high-dose regimen is expected to introduce a faster loading schedule for treatment-naïve patients.
Read more: www.neurologylive.com/view/fda-app...
#Neurology #FDA #SMA
FDA Approves Higher Strength, More Effective Nusinersen Dose for Spinal Muscular Atrophy | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
The FDA has approved a higher-dose regimen of nusinersen for spinal muscular atrophy, supported by DEVOTE data showing improved motor outcomes, reduced neurodegeneration markers, and a safety profile ...
www.neurologylive.com
March 30, 2026 at 1:25 PM