PXT3003 in CMT Type 1A - Dr. David Horn Solomon

[@michaelwizouski7317]

Wish I could have gotten on the clinical trial 10 years ago when it could have helped. I understand safety but it's a risk reward that I no longer have become the time that has passed and the progression of my disease.

[@God_is_my_savior-316]

Doesn’t sorbitol cause the nerve damage seen in diabetic peripheral neuropathy?

[@hebamohamed524]

Can you help me to know the type of my son CMT as the genetic test reveal that there is a hemizygous mutation of ATP7A gene

[@Hesam0000]

To make the long story short, even upon FDA approval, this PXT3003 will not benefit us until starting Mid-2024 and if taken by a patient no real improvement until, say, 2028 . As a patient myself I'm far more hopeful about the NIH clinic of Maryland Gene-Repair which will instantly, once and forever, cure our CMT and we can start enjoying life..

[@fredzouille]

The drug acts from the first intake... Which does not prevent the use of other approaches. Moreover, nothing is known about the effectiveness of other approaches that are in clinical trials. There's a minimum of 10-year wait until it reaches patients and it's not at all guaranteed, the clinical failure rate is enormous.

[@michaelwizouski7317]

Been waiting for so long and longer yet to still wait. Not really any hope being a 10% reduction in progression isn't going to help much with the disease already at a 80% progression. Really tired of the false hope this company has given over the last decade. Too little too late.

[@Hesam0000]

@@michaelwizouski7317 ... Pharnext won't cure us, the cure is by Gene repair and it's already available but doctors won't do it because of the fear of lawsuits if body reacts negatively.

[@michaelwizouski7317]

@@Hesam0000 just more false hope then for a therapy that won't be available until most of my youth is gone, great

[@Hesam0000]

@@michaelwizouski7317 Why do you think US doesn't have a national healthcare while doctors, pharmaceuticals and hospitals rob medicare equal to 5 to 10 times as much? Because they rather spend $10 million buying politicians than to allow national healthcare and lose $100billion easy money. If genetic repair is allowed maybe 80% of disease get cured and healthcare industry will lose billions.