¿Qué logra el nuevo fármaco que frena una ELA genética? Can Ruti estrena tofersen para ELA SOD1: punción lumbar a quién va dirigido y qué cambia en fuerza, respiración y supervivencia del paciente. El Hospital Germans Trias i Pujol, el Can Ruti de Badalona...

¿Qué logra el nuevo fármaco que frena una ELA genética? #11defebrero #felizmiercoles #ELA #EsclerosisLateralAmiotrofica #Tofersen #Qalsody #Sanidad #Salud #Neurologia #InvestigacionMedica #EnfermedadesRaras #Barcelona #Catalunya #CanRuti #MedicinaDePrecision donporque.com/nuevo-farmac...

First treatment approved in the UK for rare genetic form of MND - UKMNDRI The MHRA has approved the use of tofersen, the first licensed treatment in the UK for MND caused by a variation in the SOD1 gene.

The first treatment for a form of #MND/#ALS has been approved in the UK.

#Tofersen is authorised for SOD1 MND, demonstrating the potential of gene-targeted therapies.

UKMNDRI RI partners @sheffielduni.bsky.social & @kingsioppn.bsky.social contributed to the trials.

ukmndri.org/2025/07/31/f...

Regulatory and Clinical Outcomes of Nononcology Accelerated Approvals This study uses US Food and Drug Administration (FDA) data to characterize pivotal and confirmatory trials supporting agency decision-making in the context of the FDA accelerated approval pathway for ...

such as #aducanumab for #Alzheimerdisease, #tofersen for ALS , #delandistrogene #moxeparvovec for #Duchennemusculardystrophy & #voxelotor for #sicklecelldisease
@jama.com

ja.ma/3GHGhDE

jamanetwork.com/journals/jam...

#MND #Tofersen #PrescribeLife

Over 19,000 people have already signed our #Tofersen petition - but we're trying to get still more support for people with SOD1 #MND.

Here's a flyer that can be printed and taken to @mndassoc.bsky.social and other events to encourage even more people to support the petition 👇 #Prescribelife

#MND #tofersen #prescribelife

Why we’re campaigning on tofersen again Tofersen is a new treatment that is effective for the roughly 2% of people with MND who have a particular genetic change.

Why is #tofersen our number one #mnd campaign right now? Read on to find out more and take action! #prescribelife

www.mndassociation.org/media/latest...

#MND #Tofersen #Prescribelife

11 reactions | "Every day I wait. More of my independence is lost." Watch Seckin McGuirk, who is living with SOD1 MND, speak about the importance of accessing... | By MND Campaigns | Facebook "Every day I wait. More of my independence is lost." Watch Seckin McGuirk, who is living with SOD1 MND, speak about the importance of accessing...

"Every day I wait. More of my independence is lost."

Watch Seckin McGuirk, living with SOD1 #MND, speak about the importance of accessing #tofersen.

www.facebook.com/mndcampaigns...

And if you haven't already, please sign the #Prescribelife petition here: act.mndassociation.org/2025-prescri....

Sign our petition to help people living with SOD1 MND access a life-changing treatment Sign our petition to help people living with SOD1 MND access a life-changing treatment Seckin is among the 2% of people with MND whose disease is caused by a fault in the SOD1 gene; and for them, ther...

I’ve just signed a petition to ask the Government to make sure people with the SOD1 type of #MND can access #tofersen, a life-saving treatment. Can you sign too? act.mndassociation.org/2025-prescri...

Sign our petition to help people living with SOD1 MND access a life-changing treatment Sign our petition to help people living with SOD1 MND access a life-changing treatment Seckin is among the 2% of people with MND whose disease is caused by a fault in the SOD1 gene; and for them, ther...

I’ve just signed a petition to ask the Government to make sure people with the SOD1 type of #MND can access #tofersen, a life-saving treatment. Can you sign too?
act.mndassociation.org/2025-prescri...

Thank you Tonia Antoniazzi - not the response we'd hoped for from UK Government but we'll keep calling for everyone who needs it to have access to tofersen.

Sign the petition here: act.mndassociation.org/2025-prescri...

#tofersen #MND #Prescribelife

We made it - onwards to 7,000! #mnd #tofersen #prescribelife

Less than 150 signatures to go till we get to 6,000 names supporting access to #tofersen - thank you so much for all your help!

Let's get this petition to the next milestone #mnd #prescribelife

Sign our petition to help people living with SOD1 MND access a life-changing treatment Sign our petition to help people living with SOD1 MND access a life-changing treatment Seckin is among the 2% of people with MND whose disease is caused by a fault in the SOD1 gene; and for them, ther...

I’ve just signed a petition to ask the Government to make sure people with the SOD1 type of #MND can access #tofersen, a life-saving treatment. Can you sign too? act.mndassociation.org/2025-prescri...

Sign our petition to help people living with SOD1 MND access a life-changing treatment Sign our petition to help people living with SOD1 MND access a life-changing treatment Seckin is among the 2% of people with MND whose disease is caused by a fault in the SOD1 gene; and for them, ther...

I’ve just signed a petition to ask the Government to make sure people with the SOD1 type of #MND can access #tofersen, a life-saving treatment. Can you sign too? act.mndassociation.org/2025-prescri...

Grande nouvelle! Des recherches cliniques menées au Neuro ont permis de valider une thérapie révolutionnaire qui peut ralentir la progression de la #SLA de type #SOD1. #SantéCanada a accordé une approbation conditionnelle au #Tofersen maintenant appelé #Qalsody. Entrevue: bit.ly/qalsodyurc

Montreal research results in ‘breakthrough’ drug for ALS Clinical trials for a new ALS drug took place at the Neuro Hospital in Montreal and the results are being called a 'breakthrough.'

*This* is why clinical research is vital! A trial run at the CRU led to a breakthrough therapy that allows individuals with #SOD1 #ALS to stabilize their disease and even regain function. #Qalsody has now received conditional approval from Health Canada. #raredisease #tofersen
bit.ly/qalsodyhcan

I've signed the #PrescribeLife petition. Will you sign too?
Motor neurone disease (#MND) is fatal. But #tofersen, a groundbreaking treatment, could change that for some people. We want it to be given the best chance of being made available on the NHS. mndassoc.org/40jVN03

Breakthrough drug for SOD1 MND takes a crucial step forward - UKMNDRI Tofersen, a drug shown to slow the progression of SOD1-MND, is closer to approval following a successful campaign by the MND community. Motor neuron disease (MND) is a progressive neurological condi...

RESEARCH NEWS🚨 #Tofersen, a drug shown to slow progression of #SOD1-MND, is closer to approval in the UK. NICE has reconsidered its evaluation process after calls from the #MND community, offering renewed hope.

Read more here: ukmndri.org/2024/11/21/b...