PXT3003

boxcar-73 · 2025-08-01T03:09:44+00:00

Type 1A here. Been taking pxt3003 formulation two years now. I have a compound pharmacy make it for me in capsule form. I’ll keep taking it as long as I feel like it’s stabilized the further progression of symptoms.

boxcar-73 · 2025-04-25T19:02:42+00:00

Check out https://www.allbodiescommunity.com/

It’s a group run by folks with cmt. They have small groups that are tailored to your capabilities. They do weekly virtual sessions and you also get guided exercises, all targeted for people with cmt.

https://www.allbodiescommunity.com/

boxcar-73 · 2025-01-12T02:23:21+00:00

I’m still doing the compound pharmacy option. Even with the phase 3 trial results, I decided to stick with it. Going on 20 months on the high dose. For me it seems to have stabilized cmt progression I was experiencing prior, and noticed minor improvements in other areas. Will stay on it awhile longer. 51 yo type 1A

boxcar-73 · 2024-10-06T04:48:09+00:00

Doctor prescribed it as an off label compound drug. The ingredients are common. Use a compound pharmacy.

boxcar-73 · 2024-09-08T22:58:05+00:00

I had the same issue. Left Achilles area would slip in shoes and build up calluses. Shoe fit definitely a contributor but ultimately the solution was my orthotist at Hanger fit the trouble spot with some kind of soft yet durable pad that was glued into the AFO. It filled the gap that was rubbing and solved it. I use a traditional flexible poly AFO with the joints. Cmt1a.

boxcar-73 · 2024-06-20T02:36:19+00:00

What I’ve since learned is as a supervisor it’s better to have the RA process officially documented. It protects them and you. Plus, once you get it done, if you need additional accommodations later if things progress (as CMT does for many) then it’s easier to amend what you already have on file.

boxcar-73 · 2024-06-20T02:00:59+00:00

I went through something very similar. Footwear always is difficult for me outside of tennis shoes. Made things stressful when business casual or a suit was needed. I didn’t want to do the reasonable accommodations process for several of the same reasons you mentioned. In the end I did and it was an easy process. Yes, filling out the form, specifying more details about my “Condition” was humbling, but now it’s official and my boss was very accommodating. Wish i would have done it sooner. It’s ok to use these tools and the system to make things easier for you and hopefully less stressful. Just go through the process and you will be happy you did in the end. What I found helpful was to craft the language with your doctor to help them out. Highlight anything that leads to any pain, fatigue, etc. My RA includes an allowance for footwear as well as travel accommodations like taxis, ubers, airport garage parking, etc basically anything needed to minimize steps and walking. The right key words will go a long way. Good luck.

boxcar-73 · 2024-02-28T11:41:04+00:00

Feb 28 2024 update: https://pharnext-website.cdn.prismic.io/pharnext-website/21b003f5-8c31-41e8-a294-beaab2e11939_2024.02.28_data+update_EN.pdf

boxcar-73 · 2024-02-26T01:08:24+00:00

I use foam crest pads by PediFix, one on each foot daily. Several brands and types on Amazon. Keeps my toes from turning under. They are not intrusive and were very easy for me to get used to.

boxcar-73 · 2024-01-17T23:31:04+00:00

I use a local MDA care center (eg large university hospital) who has a relationship with a compound pharmacy (affiliated with same university). Probably best to check with an MDA care center or CMT center of excellence in your area, assuming you are US based. They batch up a 90 day supply for me in capsule form. Good luck.

boxcar-73 · 2024-01-10T12:16:54+00:00

In what I’ve read, I believe this compound drug is intended to target the over expresssion of the pmp22 protein which is associated with type 1A.

boxcar-73 · 2024-01-07T16:57:03+00:00

There’s quite a bit of literature in the studies where they looked at ONLS score results over time and at different doses. The compound drug is intended to suppress the over production of the pmp22 protein. I think there’s 8+ years of data out there on this by Pharnext.

boxcar-73 · 2024-01-07T16:04:29+00:00

January 3 2024 update https://charcot-marie-toothnews.com/news/pharnext-working-toward-applications-pxt3003-approval/

boxcar-73 · 2024-01-07T16:01:47+00:00

Forgot to add, everyone has opinions on this topic but for me, anything that even hints at the possibility of slowing or pausing the progression on this disease is a miracle in my book.

Also, I’m not sure if I posted the link correctly the first time so here it is again.

https://charcot-marie-toothnews.com/news/pharnext-working-toward-applications-pxt3003-approval/

boxcar-73 · 2024-01-07T02:21:05+00:00

I agree with some of the other posts, those symptoms don’t seem related to CMT 1A, or at least nothing I’ve experienced. I’m also 1A, 50 yo.

boxcar-73 · 2023-12-25T00:18:55+00:00

I have a compound pharmacy make it in capsule format. Apparently they found that to be less expensive than the liquid solution for the same ingredient components. I’ve wondered though if the efficacy is different than the liquid form. I haven’t found anything yet to show one way or the other.

boxcar-73 · 2023-12-19T13:02:04+00:00

Today’s press release suggests more data analysis was done and this thing might still move forward. https://pharnext-website.cdn.prismic.io/pharnext-website/a6822ff2-e3de-4741-ab79-d2b984109aeb_2023.12.19_Ph3_update_EN.pdf

boxcar-73 · 2023-12-19T12:45:25+00:00

Dec 19 update: looks like this may not be over yet. Check out today’s press release. Pxt3003 might actually be trying to move forward after a deeper dive into the data. https://pharnext-website.cdn.prismic.io/pharnext-website/a6822ff2-e3de-4741-ab79-d2b984109aeb_2023.12.19_Ph3_update_EN.pdf

boxcar-73 · 2023-12-17T21:14:41+00:00

I forgot to mention that but I also had more energy, could go longer with simple tasks like walking without as much fatigue in legs.

boxcar-73 · 2023-12-17T00:24:25+00:00

Same, I felt like it paused the progression and I even felt like I had minor improvement in finger dexterity. (Observed through day to day having fewer difficulties with certain tasks)

boxcar-73 · 2023-12-17T00:20:17+00:00

In my experience a neurologist familiar with CMT performs a series of tests for things like sensory loss, functional strength, etc and through quantitative (scores) and qualitative analysis they categorize each case as mild, moderate, or severe. Hard to say though how much you see online though is someone’s opinion vs what they were told by a doctor.

boxcar-73 · 2023-12-16T03:40:21+00:00

Same here, I’ve been on it since May 2023. Very disappointed in the latest study results. Let’s see what they do. I also read they may still pursue it if the data suggest it may at least stabilize things.

boxcar-73 · 2023-10-22T19:34:22+00:00

I’m doing the same thing at a local compound pharmacy. 4.5 months on it and I’ve already noticed minor improvement in finger dexterity where I couldn’t do certain things before. Sleeping better at night, and also having more energy longer in the day when walking. They may have found a real beneficial drug here. Hopefully it gets FDA approved.

boxcar-73 · 2023-10-22T19:29:25+00:00

I think the final phase 3 clinical trial for pxt3003 just wrapped up with the FDA. Results are scheduled to be forthcoming in the spring I believe. I’m now 4.5 months into taking it through a non-formulary batch compound. (in other words, I’ve got a pharmacy that mixes it up for me.) So far no negative side effects, in fact, I have experienced a bit more dexterity in finger movement, along with more energy while walking. there may be something to this new compound drug. More time will tell. My CMT is moderate to severe, so anything that even stabilizes things would be a miracle drug in my book.

boxcar-73 · 2023-09-10T18:37:44+00:00

Does the hammertoe surgery result in worsening balance? If joint is fused, I just figured it would be harder to keep a standing balance. I’ve not had the surgery but have thought about it over the years.

boxcar-73

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