We are so excited to open registration to our summit next week and debut some of the amazing talks and speakers we have lined up. And we are excited that ALS Network is helping be our local host. #EndtheLegacy #GeneticALS #GeneticFTD #c9orf72 #sod1 #fus

We are coming together in Sacramento in March, following other summits in North America in 2024 and 2025. We hope you consider joining us September 24th - 26th in Sacramento CA with local host ALS Network. Registration opening later this month. #EndtheLegacy #c9orf72 #grn #fus #sod1 #tardpb #mapt

🎨Science Visuals |🧠Amyotrophic Lateral Sclerosis (#ALS) is a progressive #neurodegenerative disease marked by the loss of upper & lower motor neurons.
🔬Risk factors: genetic mutations (e.g. #SOD1, #C9orf72, #TDP-43) and environmental triggers (e.g. smoking, pesticides, and heavy metals).

If you are gene negative and want to act as a control you can reach the ALL ALS study in the US at https://www.all-als.org/ if you are at risk and don't know your status , or you know you are positive see studies recruiting here https://www.endthelegacy.org/recruiting-studies
#c9orf72 #EndtheLegacy

BioRxiv Article

[BioRxiv] Therapeutic modulation of the calpastatin/calpain pathway restores calpain-mediated synaptic proteolysis and preserves motor neurons survival and function in C9orf72 ALS

https://doi.org/10.64898/2025.12.08.692907

#neurodegeneration #biorxiv #C9ORF72 #ALS #Neurodegeneration

Brain metabolic connectivity in ALS due to C9ORF72 hexanucleotide expansion: a [18F]FDG-PET study - PubMed In the comparison with ctrl-ALS, C9-ALS showed a relatively lower metabolism in the thalami and a relatively higher metabolism in the brainstem and the cerebellum. As compared to ctrl-ALS, C9-ALS show

Brain metabolic connectivity in ALS due to C9ORF72 hexanucleotide expansion: a [(18)F]FDG-PET study.

https://pubmed.ncbi.nlm.nih.gov/41379346/

#neurodegeneration #pubmed #C9ORF72 #ALS

In a new study, Masrori et al. (2025), published in *Nature Neuroscience*, researchers looked at how the C9orf72 gene, which is linked to ALS, affects brain cells called microglia.

#ALS #neuroscience #C9orf72

Check out the video to know more.

youtu.be/8gxbHZIKLVw

BioRxiv Article

[BioRxiv] Proteomic signature and mesenchymal stromal/stem cell-derived extracellular vesicle treatment of iPSC motor neurons from different ALS patient groups

https://doi.org/10.1101/2022.07.05.498816

#neurodegeneration #biorxiv #ALS #C9ORF72

Registry Data Presented at the NEALS Consortium - FTD Disorders Registry Registry data is helping to bridge the gap between the FTD and ALS research communities and to accelerate recruitment for future clinical trials.

Data were shared at the NEALS Consortium Annual Meeting by Registry Director Carrie Milliard, showing how the Registry helps bridge FTD and ALS research and speed up clinical trial recruitment.

ftdregistry.org/press/regist...

#FTD #ALS #C9orf72 #ClinicalResearch #NumbersHavePower

We look forward to seeing continuing investigations as to what causes this difference in impact. See more at endthelegacy.org/c9orf72info

#endthelegacy #c9orf72

Check out this story in Biospace profiling End the Legacy co-founder and current Coya team member Daniel Barvin.
www.biospace.com/drug-development/coya-ex...

#endthelegacy #c9orf72 #coya

Some big updates on issues important to the C9orf72 Community dropped last week. We collected them here :https://www.endthelegacy.org/latestnews . We thank the investigators and funders of these trials and participants!

#c9orf72 #ALS #news

As promised , linked is our written report for the Genetic ALS and FTD community from NEALS 2024. We will be providing 4 sections with the first debuting today. To read the analysis along with the list visit: https://www.endthelegacy.org/neals-report-2025 #endthelegacy #c9orf72 #sod1 #nealsmtg

The webinar from MND researchers originally scheduled for this Wednesday, has been moved to the following day Thursday October 16th at 9:30am US Pacific. www.endthelegacy.org/event-details/an-update-...

#c9orf72 #EndtheLegacy

We are proud to announce our first Community Science Liaison Fellow is the amazing Yentli Soto Albrecht, PhD. Congratulations Dr. Soto Albrecht!

See more here: https://www.endthelegacy.org/communitysciencefellowship

#EndtheLegacy #c9orf72 #research #geneticALS

We are thrilled to announce two new clinics have been appointed as recognized Centers of Care for those at risk of Genetic ALS and FTD!
Information on the centers can be found on our website: https://www.EndtheLegacy.org/care

#EndtheLegacy #GeneticALS #GeneticFTD #C9orf72

Le Dr Maria del Mar Amador est neurologue au Centre SLA de Paris des hôpitaux universitaires de la Pitié-Salpêtrière à Paris.
www.endthelegacy.org/event-details/une-presen... #endthelegacy #sla #c9orf72 #sod1

Nous avons le plaisir de vous annoncer que notre première réunion en français aura lieu le jeudi 25 septembre à 18h, heure de Paris.

Si vous souhaitez pouvoir vous connecter, il faut adresser un mail avec votre demande au : info@endthelegacy.org

#c9orf72 #sod1 #endthelegacy #als #ftd #sla

We have been busy this summer of 2025! Read about it here endthelegacy.org/newsletters - including that our Pacific Region Summit is starting in a few hours - cheers to our volunteer planning committee! #c9orf72 #s0d1 #grn #fus #EndtheLegacy

The ALS and FTD fields and those running trials must heed the growing consensus that presymptomatic C9orf72 expansion carriers can be included under certain biological readings in trials where the main test is in symptomatic patients. Learn more https://www.endthelegacy.org/latestnews #c9orf72

Today we are celebrating our co-founder and Genetic ALS champion Daniel Barvin for being designated an "ALS Hero" by the largest ALS organization in the United States, the ALS Association. From the entire Genetic ALS and FTD Community we cheer you Daniel! #C9orf72 #ALShero #Leadership

Aberrant splicing exonizes C9orf72 repeat expansion in ALS/FTD - Nature Neuroscience By selectively isolating and sequencing the rare RNA transcripts containing C9orf72 repeat expansion from ALS–FTD neurons, the authors uncover an alternative splicing mechanism that explains the reten...

Excited to see the work by @Yale_INP student Suzhou Yang w/ collabs @yaleneuro.bsky.social iPSC core @jdpereira.bsky.social and @mayoclinic.org @gendron_tania on #C9orf72 #ALS & #FTD finally out in @natneuro.nature.com! @wutsaiyale.bsky.social @kavliatyale.bsky.social www.nature.com/articles/s41...

We are proud to share our co-founder and current Executive Director Jean Swidler was able to work on this recently accepted article on C9orf72 prevention trials. Cheers to the authors and funders.
#EndTheLegacy #c9orf72 #prevention
https://doi.org/10.1093/brain/awaf290

Neurodegenerative disease in C9orf72 repeat expansion carriers: population risk and effect of UNC13A The C9orf72 hexanucleotide repeat expansion is associated with ALS and certain forms of dementia, though its exact penetrance remains unclear. In a UK popu

New paper out from Jiali and team. Using @ukbiobank.bsky.social data, we quantified how #ALS and #dementia incidence increase with age in carriers of #C9orf72 expansion, and demonstrated that a common #UNC13A variant nearly doubles disease risk. #ALS #MND #FTD. academic.oup.com/brain/articl...

High-throughput screen of 100 000 small molecules in C9ORF72 ALS neurons identifies spliceosome modulators that mobilize G4C2 repeat RNA into nuclear export and repeat associated non-canonical tran...

https://pubmed.ncbi.nlm.nih.gov/40207633/

#neurodegeneration #C9ORF72

A neuroprotective role of polyunsaturated fatty acids in C9orf72-ALS/FTD.

https://pubmed.ncbi.nlm.nih.gov/40140608/

#neurodegeneration #C9ORF72 #neuroprotective

C9ORF72 poly-PR disrupts expression of ALS/FTD-implicated STMN2 through SRSF7.

https://pubmed.ncbi.nlm.nih.gov/40140908/

#neurodegeneration #C9ORF72

Also contributing were Beatrix with her poster on long-read sequencing of #C9orf72 HRE and @lizziedellar.bsky.social with her poster on #ExtracellularVesicle -based biomarker discovery in CSF.

Congratulations to @bvahsen.bsky.social on receiving the German Society for Muscular Diseases Junior Research Prize for his work on microglia in #C9orf72 #ALS! ⭐ Read more about his work here: www.nature.com/articles/s41...

Photo outside Senator Schiff's office with a staff person we met with.

Photo outside Senator Padilla's office after meeting with a staff person.

Cheers to the organizers and all who participated. The next day we were proud to partner with ALS Network for visits to the CA Senate delegation. We must have our communities voice heard wherever it needs to be! #GeneticALS #GeneticFTD #ALS #FTD #C9orf72 #SOD1 #GRN #EndtheLegacy