PRAX-944 Demonstrates Functional Improvement in ET Patients
In May 2022, Praxis Precision Medicines announced positive results from their PRAX-944 Phase 2a study. The primary objective of the study was to demonstrate safety and efficacy of the PRAX-944 compound in the treatment of essential tremor.
In the study, treatment with PRAX-944 resulted in clinically meaningful improvements in function, which were supported by improvements in tremor severity. The study included an open-label period, in which all participants were treated with PRAX-944; the results demonstrated an average improvement of 42% in the Modified Activities of Daily Living (ADL) score as compared to ADL scores prior to treatment with the PRAX-944 compound.
“The data from the PRAX-944 Phase 2a study offer hope to people living with essential tremor, for a treatment developed specifically for ET,” said Bernard Ravina, M.D., Chief Medical Officer at Praxis.
Praxis is currently running a Phase 2b study evaluating the safety of the PRAX-944 compound. This study is called the Essential1 Study and it is enrolling new patients. If you or someone you know is interested in learning more about participating, go online to schedule a call with a Clinical Trial Patient Navigator.
Running 100 Miles for ET Awareness
Most people could not imagine taking on the challenge of running 100 miles. But Peder Nelson has signed up twice!
In the fall of 2023, this Sellersburg, IN resident will participate in his second Cloudsplitter 100 ultra-marathon. Cloudsplitter takes place in the heart of Central Appalachia in the Cumberland Mountains of Virginia. It’s described as “one of the more physically demanding trails in the East.” Peder will be running to raise awareness to essential tremor and to support the mission of the International Essential Tremor Foundation.
Peder talks about his goals, how he is preparing for this run and shares his personal essential tremor story in our latest Talking Essential Tremor podcast, Running 100 Miles for ET Awareness.
In 2019, Peder attempted his first Cloudsplitter with the goal of raising $10,000 for the IETF. He fell short when he suffered ankle injuries during the race. Nonetheless, he raised a significant amount: more than $4,300.
Follow Peder’s story and journey on his Difficult Destinations Facebook page.
Be Our Newest Trailblazer
ET Trailblazers are dedicated to finding answers and lending critical support to the ET community. Their generous monthly contributions allow us to respond quickly to grant and research opportunities and develop helpful programs and outreach services.
Please join the Trailblazers today by becoming a monthly donor to the IETF. It’s convenient for you because you won’t have to worry about initiating an annual donation, and you’ll never miss an issue of Tremor Talk magazine. Plus, you’ll be making a significant and ongoing impact on the lives of people coping with ET.
Sign up today!
Is this available for patients who have received a brain stimulator?
Unfortunately, no. It’s listed in their EXCLUSION CRITERIA:
Has had prior magnetic resonance-guided focused ultrasound or surgical intervention for ET such as deep brain stimulation or thalamotomy.
Do I need to sign up to become a Trailblazer since I make a big donation and many others throughout the year? Elissa and Daniel Kline
We appreciate your donations and your involvement. We set this up to allow people the convenience of an automatic donation each month. If you would like to set this up, you are sure welcome to do so. It’s however you prefer to make your donations.
Praxis website does not allow me to schedule a call with an evaluator even though I filled out the application. Please advise.
It means someone will follow up with you.
Hello,
I have attempted to receive a questionnaire to see if I am eligible for the Plexis trial for Essential Tremor. I entered the information asked for but when I click to proceed to get the questionnaire my screen does not go to the next page.
Thank you, Fred Martinez
Could you please send me an upated overview of the history and content of the history and treatments of Tremor gram? Much appreciated.
Have the doctors started prescribing PRAX – 944 for patients with essential tremors? My mother is 87 years old.
No. Praxis is currently recruiting participants for another clinical trial for this medication.
I am very interested to join the program. I suffer from Essential Tremor for the last 19 years. I live in Venezuela and are 73 years now. What part of the states is the program taken place. I’m a volunteer for any help to this condition
Are you asking about the clinical trial for PRAX-944 or were you referring to becoming a member of our ET Trailblazers?
iI am unable to schedule a call.
We have notified Praxis and someone will be following up with you.
thank you
Hi
I’m very interested in the clinical trials for Prax-944, but can’t communicate with you. Can someone please call me at 858=232=0089 to talk about this??
I’m 82 years old and have had ET for several years.
Thanks
Sylvia
We have passed your contact information along to Praxis so they can connect with you. Thank you for your interest in participating in clinical trials.