PMP22

CMT Research Foundation and Nanite Inc. Announce Partnership to Enhance Therapeutic Efficacy of Antisense Oligonucleotides in CMT1A

Retrieved on: 
Wednesday, February 22, 2023
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As part of the partnership agreement, Nanite will design delivery vehicles for genetic medicines to Schwann cells via PNPs, an emerging technology for drug delivery to hard-to-hit targets.

Key Points: 
  • As part of the partnership agreement, Nanite will design delivery vehicles for genetic medicines to Schwann cells via PNPs, an emerging technology for drug delivery to hard-to-hit targets.
  • This makes Schwann cells the primary target of genetic therapies in most cases of CMT.
  • The safe delivery of medicines to the Schwann cells of patients with CMT1A, would mark a dramatic step forward in the potential of gene therapies for CMT disease.
  • The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT.

Pharnext: Findings on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A From ‘Real-World’ Digital Lifestyle Study, CMT&Me, to be Presented at the American Association of Neuromuscular & Electrodia

Retrieved on: 
Thursday, September 15, 2022
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Pharnext: Findings on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A From Real-World Digital Lifestyle Study, CMT&Me, to be Presented at the American Association of Neuromuscular & Electrodia

Key Points: 
  • Pharnext: Findings on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A From Real-World Digital Lifestyle Study, CMT&Me, to be Presented at the American Association of Neuromuscular & Electrodia
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • The conference is taking place from September 21-24, 2022, in-person and with an option to participate virtually, at Gaylord Opryland Resort & Conference Center in Nashville, Tennessee.
  • Charcot-Marie-Tooth (CMT) disease encompasses a heterogeneous group of inherited, severe, debilitating, progressive and chronic peripheral neuropathies.
  • The genetic mutation responsible for CMT1A is a duplication of the PMP22 gene coding for a peripheral myelin protein.

Pharnext: Findings From ‘Real-World’ Digital Lifestyle Study, CMT&Me, on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A Published in the Journal of Clinical Neuromuscular Disease

Retrieved on: 
Wednesday, September 14, 2022
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Pharnext: Findings From Real-World Digital Lifestyle Study, CMT&Me, on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A Published in the Journal of Clinical Neuromuscular Disease

Key Points: 
  • Pharnext: Findings From Real-World Digital Lifestyle Study, CMT&Me, on Symptom Burden of Charcot-Marie-Tooth Disease Type 1A Published in the Journal of Clinical Neuromuscular Disease
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • The findings on patients with the 1A subtype of CMT (CMT1A) showed that patient-reported symptoms burden is high, with study participants registering difficulties using limbs, fatigue, pain, and impaired quality of life.
  • Burden severity appears to differ across the population, possibly driven by differences in rehabilitative and prescription-based interventions, and country-specific health care variability.
  • The article, titled, Patient-Reported Symptom Burden of CharcotMarieTooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study can be accessed online here .

Pharnext Announces First Patient Enrolled in Open Label Extension of the Pivotal Phase III Study of PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A, the PREMIER Trial

Retrieved on: 
Monday, September 12, 2022
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Pharnext Announces First Patient Enrolled in Open Label Extension of the Pivotal Phase III Study of PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A, the PREMIER Trial

Key Points: 
  • Pharnext Announces First Patient Enrolled in Open Label Extension of the Pivotal Phase III Study of PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A, the PREMIER Trial
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • The dose of PXT3003 tested in the PREMIER trial corresponds to the HD tested in the prior Phase III clinical study, the PLEO-CMT trial, and the ongoing open-label extension Phase III study, the PLEOCMT-FU trial.
  • The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose (HD) tested in the prior Phase III trial (PLEO-CMT).
  • An international pivotal Phase III study of PXT3003 in CMT1A, the PREMIER trial, is currently ongoing.

Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A

Retrieved on: 
Monday, May 30, 2022
Sorbitol, Charcot–Marie–Tooth disease, Disease, Running, ISIN, Wheelchair, Patient, Walking, ClinicalTrials.gov, PMP22, Treatment, Phase II, Neurodegeneration, Life, Baclofen, Naltrexone, Safety, Occupational therapy, Risk, CMT, CET, Therapy, Muscle atrophy, Regulatory News Service, Prevalence, Schwann cell, Completion, Muscle, Pharmaceutical industry, Security (finance)

Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A

Key Points: 
  • Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose (HD) tested in the prior Phase III clinical study, the PLEO-CMT trial, and the ongoing open-label extension Phase III study, the PLEOCMT-FU trial.
  • For more information on the PREMIER trial please refer to the About the PREMIER Trial paragraph below.
  • The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose (HD) tested in the prior Phase III trial (PLEO-CMT).

New Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time

Retrieved on: 
Monday, May 16, 2022
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The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.

Key Points: 
  • The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.
  • All randomized CMT1A patients who completed the PLEO-CMT trial (treated with PXT3003 or placebo) were eligible to pursue treatment with PXT3003 in the PLEO-CMT-FU trial.
  • This trial enrolled a total of 187 patients and was designed to primarily assess the long-term safety and tolerability of PXT3003.
  • Patients treated with placebo in the PLEO-CMT trial were randomized in PLEO-CMT-FU to receive LD or HD of PXT3003.

New Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time

Retrieved on: 
Monday, May 16, 2022
Sorbitol, Disease, HD, PMP22, Lead, Naltrexone, Standard of care, Risk, High-dose chemotherapy and bone marrow transplant, CMT, Safety, Muscle atrophy, Prevalence, LD, Degenerative disease, Walking, ClinicalTrials.gov, Neurodegeneration, Schwann cell, Charcot–Marie–Tooth disease, Running, Doctor of Philosophy, Patient, Human, Occupational therapy, Therapy, MD, ALS, Data analysis, ISIN, Wheelchair, Phase II, Life, Baclofen, CET, Muscle, Pharmaceutical industry

The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.

Key Points: 
  • The entire CMT community is hopeful that PXT3003 could be the first approved therapy for this debilitating disease.
  • All randomized CMT1A patients who completed the PLEO-CMT trial (treated with PXT3003 or placebo) were eligible to pursue treatment with PXT3003 in the PLEO-CMT-FU trial.
  • This trial enrolled a total of 187 patients and was designed to primarily assess the long-term safety and tolerability of PXT3003.
  • Patients treated with placebo in the PLEO-CMT trial were randomized in PLEO-CMT-FU to receive LD or HD of PXT3003.

Pharnext: Data from First Phase III Clinical Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A, the PLEO-CMT Trial, Published in the Orphanet Journal of Rare Diseases

Retrieved on: 
Monday, October 18, 2021
Human, Schwann cell, Safety, AMF, Baclofen, Disease, FDA, Sorbitol, Lead, Phase II, Orphanet Journal of Rare Diseases, CET, Charcot–Marie–Tooth disease, Patient, Mission statement, Intention, Doctor of Philosophy, URD, ClinicalTrials.gov, Life, Neurodegeneration, HD, Company, Prospectus, MD, Genomics, Drug discovery, Degenerative disease, EMA, Occupational therapy, Genta (company), Trial of the century, ISIN, OJRD, Therapy, Intelligence, CMT, Artificial intelligence, Running, Naltrexone, PMP22, III, Walking, Wheelchair, Prevalence, LD, Alzheimer's disease, Regulatory News Service, High, ALS, Muscle atrophy, Muscle, Pharmaceutical industry, Security (finance), CHARCOT-MARIE-TOOTH DISEASE TYPE 1A ('CMT1A'), PXT3003, THE PLEO-CMT TRIAL, THE PLEO-CMT-FU TRIAL, THE PREMIER TRIAL, PHARNEXT

Pharnext: Data from First Phase III Clinical Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A, the PLEO-CMT Trial, Published in the Orphanet Journal of Rare Diseases

Key Points: 
  • Pharnext: Data from First Phase III Clinical Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A, the PLEO-CMT Trial, Published in the Orphanet Journal of Rare Diseases
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • This extension study is still ongoing, and 130 patients are still receiving treatment with high-dose PXT3003.
  • The second international, randomized, double-blind, two-arm placebo-controlled Phase III study of PXT3003, the PREMIER trial, was initiated in March 2021 in the U.S.
  • PXT3003 has shown promising and consistent results across preclinical and clinical studies in Phase II and Phase III (PLEO-CMT and PLEO-CMT-FU).

CMTA and Addex Therapeutics Enter Collaboration to Advance GABA B PAM as Potential Treatment for CMT1A

Retrieved on: 
Wednesday, September 22, 2021
Biotechnology, Therapy, Clinical trial, Partnership, Neurodegeneration, PMP22, Janssen Pharmaceuticals, Epilepsy, Peripheral neuropathy, Dyskinesia, PAM, SIX Swiss Exchange, Nasdaq, Patient, Glutamate (neurotransmitter), American depositary receipt, Peripheral myelin protein 22, Biomarker, Allosteric regulation, GLOBE, CEO, NAM, Glutamic acid, Histology, Disease, Blepharospasm, ADX71149, Â, LHC Accelerator Research Program, IND, CMT, Research, Drug development, Drug discovery, PTSD, STAR, Glutamate receptor, GABAB, Quality of life, CMTA, Electrophysiology, Protein, Lists of diseases, L-DOPA, Pharmaceutical industry

The GABAB receptor has previously been shown to be instrumental in controlling the overexpression of Peripheral Myelin Protein-22 (PMP22) in a rat model of CMT1A.

Key Points: 
  • The GABAB receptor has previously been shown to be instrumental in controlling the overexpression of Peripheral Myelin Protein-22 (PMP22) in a rat model of CMT1A.
  • Strategic alliances with pharmaceutical partners like Addex Therapeutics support drug development efforts to deliver therapies to CMT patients.
  • We are excited to establish this partnership with Addex Therapeutics as they work to advance their PAMs to treat CMT1A, said CMTAs CEO Amy Gray.
  • Preclinical programs ongoing with Addex include GABAB PAM for CMT1A, mGlu7 NAM for PTSD, mGlu2 NAM for mild neurocognitive disorders, mGlu4 PAM for Parkinsons disease and mGlu3 PAM for neurodegenerative disorders.