Last night was brutal. Frankie was hit by a wave of grief. Hearing a 6 year old talk about death from #ALS shouldn’t happen. She asked why I couldn’t save my mom. Why the medicine (Tofersen) wasn’t made sooner. We must #EndALS

ALS hastalarına umut olacak gen tedavisi onaylandı!!!
Qalsody (Tofersen), süperoksit dismutaz 1 (SOD1) geninde mutasyona sahip amyotrofik lateral skleroz (ALS) hastalarının tedavisi için FDA onayı aldı.
#ALS #genetik #tıbbigenetikderneği

#Health accepts for review a new drug submission for #Tofersen for the treatment of a rare, genetic form of ALS
#SOD1 - ALS is a devastating, uniformly fatal, and ultra-rare genetic form of #ALS that represents approximately two percent of all ALS cases

The best way to end ALS is for all of us to get genetic testing. If you do, it will unlock new treatments like Tofersen.

If you have any questions or have not yet had testing, Synapticure is here for you.

Visit Synapticure.com to learn more.

$ARWR after $IONS took some cheap shots at #RNAi + $ARWR in particular y'day, Arrowhead not only will obliterate Ionis in ApoCIII (primary outcome trig lowering), but has now demonstrated in preclinicals that #tofersen doesn't stand a chance.

Very impressive CSF-Nfl reduction.

Breaking news - Tofersen approved by EMA for use in #ALS #MND caused by SOD1 mutation (about 2% of people). UK MND Research Institute scientists including both our directors have been at the forefront, contributing to the trial and advising the EMA.
investors.biogen.com/news-releases/…

Tofersen is keeping so many of our friends alive and could have saved so many more lives if there was more access Chris Snow Kelsie Snow @sairysays Constance Marie SOD1 EndALS BridgetRebecca Lisa's Legacy for ALS Mayuri Saxena

We are honored to stand with the SOD1 community.

‼️The FDA says it encouraged the company to keep developing tofersen, but also cautioned that securing a full approval based on a single, failed trial would be challenging—JUST KIDDING!
biopharmadive.com/news/biogen-to…

FDA approves QALSODY™ (tofersen) as the first treatment targeting a genetic cause of ALS $IONS prn.to/3NeSG2P

Tofersen, or BIIB067, developed by Biogen, is a key treatment for ALS, just as Ammar Al-Chalabi details below and in the article. Sadly, the priority here is politics and economics instead of offering somebody life-changing therapeutics. #mnd #als

I am really excited for this panel as it will allow us to hear from people living with SOD1-ALS, SOD1 gene carriers, and caregivers about their experiences with Qualsody (Tofersen) and what the recent FDA approval means for them and for all of us.

RSVP at the link below.

Have you been offered blood neurofilament level monitoring? I am unsure if we have clocked just how significant the licensing of Tofersen for SOD1-ALS is for the future diagnosis and management of neurodegenerative diseases. #MS_Selfie Do you agree? => buff.ly/3V3fcNX

At 5pm ET, Synapticure goes live with patients and caregivers including Alexis Behan and @craigmelanie to share their experiences with Tofersen, the newly approved drug for SOD1 ALS that Pat has received for the last year. View his journey at 7pm on ESPN2 #NothingElseMatters Brian Wallach

Tofersen is a high-tech genetic therapy for SOD1-ALS which works. The decision by NICE to assess it like a routine #MND #ALS therapy means it is likely to be rejected for UK patients, even though it is already approved in the USA and Europe. We urge NICE to reconsider

SOD1 変異に起因するタイプの ALS の治療薬として tofersen が FDA 承認された。名称から察せられる通り核酸医薬で、変異 SOD1 の mRNA に結合して RNase H 依存性分解を惹起するアンチセンスオリゴヌクレオチド。髄腔内投与(腰椎穿刺)で、最初２週おきに３回打った後は４週おきに投与されるとのこと。

Tofersen is one of very few drugs proven to help some people with #MND #ALS . The community’s voice on this is unanimous and we support My Name'5 Doddie Foundation's pledge to take action. Please reconsider your decision, NICE

BREAKING NEWS. This is what people living with SOD1-ALS in Europe were waiting for! Biogen receives positive opinion from EMA CHMP for market authorisation of Tofersen (QUALSODY) under exceptional circumstances. #ALS / #MND is a treatable disease after all.

There are YEARS of UNPRECEDENTED #RealWorldEvidence of #NUROWN efficacy that warranted Accelerated Approval long ago for 100% FATAL #ALS .

It's important to note that Tofersen was approved by the Dr. Patrizia Cavazzoni less than 3 months ago specifically based on a significant…

For readers interested in emerging treatments for ALS, including the just-approved tofersen, here's a comprehensive recent review go.nature.com/41Ixneo