Biogen-194082 december 2022
A critical review and meta-analysis for more than 2600 SMA patients worldwide
To what extent can a treatment change the natural history outside of the clinical trial profile? How large is the impact of age, subgroup, or ambulatory status on the treatment effect?
This video is about the critical review and meta-analysis of nusinersen efficacy in type 2 and 3 pediatric and adult patients with SMA based on Real-World Evidence from 10 countries and with more than 2600 patients. Watch the presentation of Dr. Giorgia Coratti when she talks about design and the results of the publication.
Text: Biogen
Bild: Från filmen (Svensk Biogen-194082/global Biogen-156478)
Biogen-194088 december 2022
Senast uppdaterad: 2022-12-29
Referens:
Coratti G, et al. Orphanet J Rare Dis. 2021;16(1):430
Spinraza™ (nusinersen), Rx, ATC-kod: M09AX07
Injektionsvätska, lösning 12 mg, EF ej förmån.
Baserad på SPC 08/2023
Indikation: För behandling av spinal muskelatrofi av typ 5q. Dosering: Spinraza administreras intratekalt genom lumbalpunktion. Den rekommenderade dosen av Spinraza är 12 mg (5 ml) per administrering. Behandlingsschemat är 4 laddningsdoser på dag 0, 14, 28 och 63 följt av en underhållsdos en gång var 4:e månad. Biverkningar: De vanligaste biverkningarna i samband med administrering av Spinraza är huvudvärk, kräkningar och ryggsmärta. Fall av allvarlig infektion, såsom meningit, har observerats. Det har även förekommit rapporter om kommunicerande hydrocefalus, aseptisk meningit och överkänslighet (t.ex. angioödem, urtikaria och hudutslag). Kontraindikationer: Överkänslighet mot den aktiva substansen eller mot något hjälpämne. Graviditet & amning: Användning av Spinraza ska undvikas under graviditet och amning.
För ytterligare information om dosering, kontraindikationer, varningar och försiktighet, biverkningar och förpackningar se www.fass.se. Biogen-154864 oktober 2023
Contains instructions on how to perform CHOP-INTEND and how to complete the test paper
Contains instructions on how to perform HFMSE and how to complete the test paper
Contains instructions on how to perform RULM and how to complete the test paper
In just a few minutes, this PubExplainer gives you an overview of the results of the independent meta-analysis performed by Coratti et al., on real world treatment effect with nusinersen.
Dr. Bartels explains how to distinguish between perceived fatigue and fatigability and how the endurance shuttle tests can be used to assess fatigability across the SMA spectrum.
Dr. Duong explains why a novel outcome measure is needed for adult non-sitters, how it was developed using a human-centered approach and her experience of using the ATEND both in clinic and remotely.
Dr. Rodrigue reviews a Canadian outcome measure toolkit för adults with SMA. He discusses the needs unmet by the existing assessment scales and how digital assessments can be an important complement.
This short PubExplainer gives you an overview of the results of the March 29, 2019 interim analysis of the NURTURE-study in presymptomatic infants treated with nusinersen by De Vivo et al.
This PubExplainer, on a publication by Hagenacker et al., provides an overview of one of the largest and most diverse real-world studies in adult SMA-patients. The patients were followed at 10 centers in Germany.
This PubExplainer, by Maggi et al., provides an overview of one of the largest real-world studies in adult SMA-patients published so far. The patients were followed at 18 centers in Italy.
With the growing body of evidence and an evolving treatment landscape, the classification of subtypes needs a revision and Real-World Evidence becomes more and more important.
This presentation was hosted by Anna-Karin Kroksmark during 2020's Listen & Learn meeting. It was a break-out session intended for physiotherapists on habilitation and rehabilitation in SMA.
Informationsfilm om hur Spinraza (nusinersen) administreras