Great session this morning on enhancing the delivery of oligonucleotide therapeutics. #MDAConference

Important perspective on Charcot-Marie-Tooth from George Simpson. medium.com/@georgehsimpso…

Find out why we think the end of CMT is in sight. cmtrf.org/five-reasons-w…

Learn more about a recently completed project utilizing PMO ASOs to lower PMP22 levels in CMT1A. cmtrf.org/shift-project-…

The CMT Research Foundation is proud to introduce the Scientific Program Committee for the 2022 Global CMT Research Convention. They are building a stimulating program that will advance the science of finding treatments and cures for all affected by CMT. cmtconvention.cmtrf.org

In CMT1A mice, miRNA targeting PMP22 and delivered via AAV9 rescues behavior, nerve structure, and nerve function. Funded by CMT Research Foundation. jci.org/articles/view/…

There are only 2 days left to secure early bird pricing for the 2022 Global CMT Research Convention. Learn more and register at cmtconvention.cmtrf.org.

#Cover: RNAi-driven gene therapy in a demyelinating peripheral neuropathy model: buff.ly/3nkjBvX Image: toluidine blue–stained femoral motor nerve from an AAV9-miR871–treated CMT1A mouse; Credit: Marina Stavrou NeurologyGeneticsCyp #Neuroscience #Therapeutics

Congratulations to Marina Stavrou, Kleopas Kleopa, Scott Harper, and the other collaborators for making the cover of Journal of Clinical Investigation! We are very proud to have been able to fund this high impact research. jci.org/132/13

We are pleased to announce that Dr. Alessandra Bolino of Ospedale San Raffaele has completed the first stage of her project testing @AcuraStem's PIKfyve inhibitor in CMT4B1. Next up: a preclinical trial in CMT4B1 mice. cmtrf.org/bolino-milesto…

Congratulations to Afrooz @Rashnonejad of Ohio State and @NationwideKids! This is a very promising project aiming to develop a single gene therapy that has the potential to work for all of nearly all people with CMT1B.

Great opportunity to work with an exciting team!

The CMT Research Foundation is excited to announce a new project for CMT1A. With funding from the CMTRF, Spanish pharmaceutical company ORYZON will test two novel HDAC6 inhibitors in CMT1A. cmtrf.org/oryzon-project…

An act of generosity and kindness continues to snowball and inspire. Thank you to CMT Country Music Television for the donation and to the anonymous donor who is matching each gift up to $250,000. Get on board now-follow the link at the end of this story. cmt.com/news/783o2g/cm…

Read more about our newly announced project with Samsara Therapeutics, an innovative biotech company targeting neurodegenerative disease with autophagy enhancers. If successful, the project will result in a lead candidate for CMT1A and data for an IND submission. cmtrf.org/samsara-therap…

The latest episode of the Biotechnology Innovation Organization podcast is up, featuring our Co-Founder and Chief Mission Officer Susan Ruediger, discussing why we founded the CMT Research Foundation and what we are accomplishing. iambio.simplecast.com/episodes/the-s…

Turn my $5,000 into $10,000 to fight CMT! For each donation through my personal fundraiser from now to the end of the year, I will personally match dollar-for-dollar up to $5,000. The end of CMT is in sight -- click the link to help make it a reality! secure.qgiv.com/event/cmtresea…

Can't wait to get started tomorrow at the LBDA !

Dear colleagues, The michaeljfox.org (MJFF) is collecting information on cohorts with Lewy body diseases, including DLB. Please consider completing this important RFI and/or forwarding through your professional networks. Lewy Body Dementias PIA forms.office.com/pages/response…

LBDA is hiring a remote Education Coordinator. Come join a dedicated and growing team! indeed.com/job/education-…