📄 New study: A novel implantable catheter–port (ThecaFlex DRx) enables repeated intrathecal delivery of nusinersen in SMA.

✔️ 100% implantation success
✔️ 92% received treatment via device
✔️ Promising early safety profile

buff.ly/Fwf37nT

#SMA #Nusinersen #Neurology

FDA Approves High-Dose Regimen of Nusinersen for Spinal Muscular Atrophy Biogen announced that the FDA has approved a high-dose regimen of nusinersen for the treatment of SMA in pediatric and adult patients.

The @fda.gov has approved a high-dose regimen of #Nusinersen (#Spinraza®) for the treatment of spinal muscular atrophy (#SMA) in pediatric and adult patients, Biogen announced.

Learn more: https://bit.ly/4s7yn8e

#RareDisease #SpinalMuscularAtrophy #MedSky

バイオジェン、ヌシネルセン高用量がNature Medicine掲載──SMAでCHOP-INTEND有意改善、米PDUFAは2026年4月3日 | STELLANEWS.LIFE STELLANEWS.LIFE（ステラニュース・ライフ）は、科学技術・医療・ライフサイエンスの分野における研究 | STELLANEWS.LIFE（ステラニュース・ライフ）は、科学技術・医療・ライフサイエンスの分野における研究

Biogen’s high-dose nusinersen published in Nature Medicine; DEVOTE shows significant CHOP-INTEND improvement in SMA

stellanews.life/technology/8...

#SMA #Nusinersen #SPINRAZA #DEVOTE #CHOPINTEND #FDA #PDUFA

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.

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

Last 4 November, Nicole Gusset, CEO of SMA Europe, took part in a press conference in Warsaw presenting nearly seven years of real-world data from Poland’s B.102 drug programme for Spinal Muscular Atrophy (SMA). 📣 #SMA #Nusinersen #SpinalMuscularAtrophy #PatientCare #RareDiseaseCommunity

A retrospective cohort study assesses bulbar function in children with #SpinalMuscularAtrophy type 1 treated with #nusinersen, finding variable improvements that underscore the need for focused bulbar monitoring
onlinelibrary.wiley.com/doi/10.1111/...

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

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...

Nusinersen Improves Motor Function in Different Types of SMA Nusinersen leads to significant clinical efficacy and favorable safety in children with spinal muscular atrophy (SMA), a study found.

#Nusinersen (#Spinraza) showed “significant clinical efficacy and favorable safety” in children with spinal muscular atrophy, in a real-world analysis. Following insurance coverage, the treatment also showed improved economic feasibility.

Read more: bit.ly/3XLLoIf

#RareDisease

But great deal for #$biib. They are ideally positioned to market this largely pediatric intrathecal oligo based on #nusinersen SMA experience. Would love to know how much they offered for entire Co. #$stok

If you missed it from last week:

The #FDA accepted a sNDA for higher dose #nusinersen (Spinraza; Biogen Inc) for spinal muscular atrophy, made up of two 50 mg doses, 24 days apart. #SMA

Additional details here 👇
www.contemporarypediatrics.com/view/fda-acc...

Michał Stankiewicz

♿️ Warto może też wspomnieć, ale nie jest to priorytet, że jestem niepełnosprawny — choruję na rdzeniowy zanik mięśni (SMA) typu II i od 2020 przyjmuję #Nusinersen (#Spinraza).
To chyba wszystko. Jakbyście chcieli zobaczyć moje projekty, to zapraszam na www.stankiewiczm.eu.
Pozdro!

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