Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A

Arthur Suckow, PhD, chief executive officer at DTx Pharma talks about Charcot-Marie-Tooth Disease Type 1A (CMT1A) and a treatment in development for this rare disease.
CMT1A is a rare inherited neurological disorder that causes damage to the peripheral nerves. It is caused by a duplication of the PMP22 gene that leads to poor myelination of axons. Current treatment options are symptomatic and may include physical therapy, occupational therapy, braces and other orthopedic devices, orthopedic surgery, and pain medications.
DTx Pharma is developing a treatment for CMT1A that reduces the duplication of the PMP22 gene. More specifically, the company is developing a method to deliver short interfering RNAs (siRNAs) that inhibit the expression of disease causing genes. The company is finalizing its preclinical studies and plans to begin clinical trials in late 2023 or early 2024.
To learn more about CMT1A, visit checkrare.com/charcot-marie-tooth-disease-type-1a/

[@justinaspetkauskas9650]

I have cmt1A please sign me up!

[@justinmayy]

How do I sign up for the clinical trial and which clinical trial is it?

[@ispk25235]

SIGN ME UP!!!!!!!!!!!!!!!!!!

[@মারান্নাঘর]

Can you please tell me what is the price ?? You give me a hope but ironically my father is poor .

[@ShuvamDadal]

Iam suffer cmt .i want to treat if possible

[@notodayorever]

How do you sign up?

[@kikkan7110]

Thank you and thank you God!

[@tyrone1son]

How do I sign my wife up?

[@chaimutyala6981]

Awesome news and research !

[@jtspiky]

whats the difference between this and the pxt3003 trials ?

[@lska7853]

How to I sign up my wife?

[@westybyrd2047]

How do I sign up?

[@markknebel8923]

I was just diagnosed with CMT on this past Thursday. I don't know the type yet. I am scheduled for another extensive EMG on both legs. I read that there is no cure. How does this work? Thanks.

[@kevinfitzgerald7474]

What is the cause of these duplicating of these genes and demyelinating of the nerves sheaths. I was told in the past to watch out for cuts so they don't get infected but I was never told lyme and environmental illnesses in long term absolutely does cause the cmt peripheral nueropothy symptom to ger worse. As I have a tick disease and environmental illness. There are causes to these nuerological disorders. It would be great to find a cure for cmt and these other nuerological problems tick diseases and environmental illnesses cause. There are stem cell therapy to treat nuerological problems. Tick diseases and environmental illnesses do cause cmt to get worse. I'm having a severely difficult time functioning. There needs to be research on how these tick diseases, Lyme and environmental illnesses cause cmt to get worse and a cure for all this. Is it right for nuerology to not help me with anything now because every specialist and nuerology are afraid of lyme disease and environmental illnesses. Im not getting any help for my cmt now. Very unprofessional.

[@walkinghorsejones]

I have cmt 1a ..lucky me 😅sign me up

[@ThomasHedde]

where are they doing this?

[@daimogu95]

I have CMT2F, if you need me, count me in.

[@jimmyhand1259]

So what's new with this?

[@DebraSwank]

What about 1X ?

[@goobahc2446]

sign me up.

[@memyselfandi36]

My wife has cmt type 1a she's wanting to participate in the trials if possible??