pattismith
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Novel Drug Cocktail Offers Rare Neuropathy Patients New Hope
— Charcot-Marie-Tooth patients see improved outcomes with repurposed drugs
by Judy George, Senior Staff Writer, MedPage TodayMay 8, 2019
The investigational combination of baclofen, naltrexone, and sorbitol known as PXT3003, given as a twice-daily oral solution, offered the first evidence of meaningful improvement for patients with Charcot-Marie-Tooth type 1A, said Florian Thomas, MD, PhD, of Hackensack University Medical Center in New Jersey, at the Emerging Science session of the 2019 American Academy of Neurology annual meeting.
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The polytherapy has multiple mechanisms of action, according to drug maker Pharnext: a synergistic inhibition of PMP22 gene overexpression associated with myelination improvement, direct nerve protection, and positive effects on muscle cells, neuromuscular junctions, and immune
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Dose 3 was 6 mg baclofen, 0.70 mg naltrexone, and 210 mg sorbitol; dose 4 was twice as much as dose 3. Both doses were given twice daily for up to 15 months.
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Over 12 to 15 months, patients in the dose 4 group improved while the "placebo [group] deteriorated, as expected,"
— Charcot-Marie-Tooth patients see improved outcomes with repurposed drugs
by Judy George, Senior Staff Writer, MedPage TodayMay 8, 2019
The investigational combination of baclofen, naltrexone, and sorbitol known as PXT3003, given as a twice-daily oral solution, offered the first evidence of meaningful improvement for patients with Charcot-Marie-Tooth type 1A, said Florian Thomas, MD, PhD, of Hackensack University Medical Center in New Jersey, at the Emerging Science session of the 2019 American Academy of Neurology annual meeting.
....
The polytherapy has multiple mechanisms of action, according to drug maker Pharnext: a synergistic inhibition of PMP22 gene overexpression associated with myelination improvement, direct nerve protection, and positive effects on muscle cells, neuromuscular junctions, and immune
.....
Dose 3 was 6 mg baclofen, 0.70 mg naltrexone, and 210 mg sorbitol; dose 4 was twice as much as dose 3. Both doses were given twice daily for up to 15 months.
...
Over 12 to 15 months, patients in the dose 4 group improved while the "placebo [group] deteriorated, as expected,"