Charcot–Marie–Tooth disease

Pharnext Announces Successful Debt Refinancing and New Loan Agreement

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星期三, 六月 8, 2022
Loan agreement, IPF, Company, CET, Charcot–Marie–Tooth disease, Neurodegeneration, Partnership, Regulatory News Service, Therapy, ISIN, Patient, Trial of the century, Risk, Convertible, BSA, EUR, Medicine, Loan, Security (finance), Pharmaceutical industry, Credit

This Loan Agreement will provide the Company with added flexibility to manage its balance sheet and funding requirements.

Key Points: 
  • This Loan Agreement will provide the Company with added flexibility to manage its balance sheet and funding requirements.
  • Dr. David Horn Solomon, Chief Executive Officer of Pharnext, commented: We are grateful to Alpha Blue Ocean for its continued support of Pharnext.
  • Alpha Blue Ocean is committed to advancing promising companies and this agreement with Pharnext supports our mission.
  • Pharnext draws the attention of investors to the financial and other risk factors detailed in its financial reports.

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

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星期一, 五月 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

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星期一, 五月 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.

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

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星期一, 五月 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: The Latest Findings From CMT&Me, a ‘Real-World’ Digital Lifestyle Study on Charcot-Marie-Tooth Diseases (‘CMTs’), to be Presented at the 2022 Peripheral Nerve Society (‘PNS’) Annual Meeting

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星期四, 五月 5, 2022
Booth, PNS, CET, Poster, Patient, Charcot–Marie–Tooth disease, Regulatory News Service, Risk, Neurodegeneration, CMT, Therapy, CMTS, ISIN, InterContinental Hotel Tamanaco, Disease, Annual general meeting, Awareness, InterContinental, Consumer electronics, Security (finance), Pharmaceutical industry

Pharnext: The Latest Findings From CMT&Me, a Real-World Digital Lifestyle Study on Charcot-Marie-Tooth Diseases (CMTs), to be Presented at the 2022 Peripheral Nerve Society (PNS) Annual Meeting

Key Points: 
  • Pharnext: The Latest Findings From CMT&Me, a Real-World Digital Lifestyle Study on Charcot-Marie-Tooth Diseases (CMTs), to be Presented at the 2022 Peripheral Nerve Society (PNS) Annual Meeting
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • The conference is taking place on May 14-17, 2022, in-person at the InterContinental Hotel, in Miami, Florida, US.
  • An international pivotal Phase III study of PXT3003 in CMT1A, the PREMIER trial, is currently ongoing.
  • Pharnext is listed on the Euronext Growth Stock Exchange in Paris (ISIN code: FR0011191287).

Joshua Schafer Appointed as Interim Chairman of Pharnext Board of Directors

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星期五, 四月 1, 2022
Alzheimer's disease, Takeda Pharmaceutical Company, CET, Therapy, Interim, Neurodegeneration, Board, Business, Program, Genomics, General manager, Patient, Medicine, Intelligence, File, ISIN, CMT, Artificial intelligence, MS, Notre Dame, Drug discovery, Autoimmunity, Regulatory News Service, University, Charcot–Marie–Tooth disease, Security (finance), Pharmaceutical industry, Biotechnology, Northwestern University, Biology, Mallinckrodt

PARIS, France, March 30, 2022, 6:00pm CET - Pharnext SA (FR0011191287 - ALPHA) (the "Company"), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY(TM) platform, announces that Joshua Schafer has been appointed as interim Chairman of the Pharnext Board of Directors with immediate effect.

Key Points: 
  • PARIS, France, March 30, 2022, 6:00pm CET - Pharnext SA (FR0011191287 - ALPHA) (the "Company"), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY(TM) platform, announces that Joshua Schafer has been appointed as interim Chairman of the Pharnext Board of Directors with immediate effect.
  • This follows the resignation of Elisabeth Svanberg from the position of Chairperson and from the Board of Directors of Pharnext.
  • Joshua Schafer joined the Pharnext Board of Directors in July 2020.
  • On his appointment as Interim Chairman of the Pharnext Board, Joshua Schafer commented: "I am pleased to have taken on the interim Chairman role at Pharnext as we advance our pivotal Phase III study of PXT3003 in Charcot Marie Tooth disease type 1A ('CMT1A'), the PREMIER trial.

The Charcot-Marie-Tooth Association Earns a Perfect Rating From Charity Navigator

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星期三, 二月 23, 2022
GLOBE, Physician, Efficiency, Charitable organization, Financial health management, 2009 Davis Cup, Charity Navigator, Budget and Accounting Transparency Act of 2014, Charcot–Marie–Tooth disease, CMTA, STAR, Peripheral neuropathy, LHC Accelerator Research Program, Biotechnology, Organic, Patient, Quality of life, Research, CEO, CMT, Medical device, Pharmaceutical industry

The CMTA is only one of 15 health-related charities to earn a perfect score from Charity Navigator, and the only CMT-related organization to earn four stars.

Key Points: 
  • The CMTA is only one of 15 health-related charities to earn a perfect score from Charity Navigator, and the only CMT-related organization to earn four stars.
  • "We are honored to be recognized by Charity Navigator with its top rating," said Amy Gray, CEO of the CMTA.
  • Charities with perfect scores earn the distinction of being recognized among Charity Navigator's "Perfect 100."
  • Less than one percent of the thousands of charities rated by Charity Navigator have earned perfect scores.

Pharnext appoints Valérie Worrall as Chief Financial Officer

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星期四, 二月 17, 2022
EOS, Charcot–Marie–Tooth disease, Patient, CET, Prospectus, URD, AMF, Company, Neurodegeneration, Informatics for Consumer Health, CFO, Regulatory News Service, ISIN, Euronext, HEC Paris, Medtech, Intention, Therapy, Genomics, Novartis, Artificial intelligence, Mission statement, BALT, Drug discovery, Intelligence, EOS imaging, Alzheimer's disease, Security (finance), Pharmaceutical industry, Pharma

Valrie Worrall is an experienced finance professional with over 25 years' experience across Pharma, Consumer Health and MedTech.

Key Points: 
  • Valrie Worrall is an experienced finance professional with over 25 years' experience across Pharma, Consumer Health and MedTech.
  • At Pharnext, Valrie Worrall will be responsible for advancing new fundraising opportunities and supporting the Company's growth.
  • Dr. David Horn Solomon, Chief Executive Officer of Pharnext, commented: "I am delighted to welcome Valrie to the team and look forward to working with her.
  • On her appointment as Chief Financial Officer, Valrie Worrall of Pharnext, commented: "I am excited to be joining Pharnext at this critical time of its development.

Muscular Dystrophy Association Gathers Global Leaders at Annual Conference, the Largest Convening in the Field of Neuromuscular Disease Research & Care in the World

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星期三, 二月 9, 2022
Molecule, Transfer, CRISPR, Industry, Duchenne muscular dystrophy, Muscular Dystrophy Association, Clinical trial, Multimedia, Gaylord Opryland Resort & Convention Center, Episcopal conference, Genetic heterogeneity, ALS, Science, Conference, Doctor of Philosophy, Convention center, Horizontal gene transfer, Session, Invention, Federation, 2021 Essex County Council election, Knowledge, Gene editing, Spinal muscular atrophy, Translation, CEO, Peripheral neuropathy, MDA, Duchenne, Chairperson, Environment, Neuromuscular disease, Telehealth, Drug development, CMT, SMA, Muscular dystrophy, IRIS, View, Degenerative disease, Amyotrophic lateral sclerosis, Charcot–Marie–Tooth disease, MPH, National Sexual Violence Resource Center, COVID-19, Pharmaceutical industry, Medical imaging, Industrial design, DMD, Gene, Research, MD

Day four of the conference will be devoted to oral presentations from selected abstracts submitted by scientific and clinical researchers.

Key Points: 
  • Day four of the conference will be devoted to oral presentations from selected abstracts submitted by scientific and clinical researchers.
  • "MDA's annual conference is the largest and most important convening on advances in understanding and treating neuromuscular diseases in the world.
  • "Our conference is the only one in the neuromuscular space that really combines everything from basic research to clinical care, treatment, and management."
  • For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases.

StandStrong Disrupts Orthotic Market With Wearable Foot Support For Women's Diverse Lifestyles

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星期五, 一月 28, 2022
CEO, CMT, Charcot–Marie–Tooth disease, Heel, Gait, Patent, Technology, Woman, Nursing, Pressure, Freedom, Pain, Time, Foot, Art in the US Women's Suffrage Movement, Shoemaking, Plantar fasciitis

Female-owned StandStrong is shaking up the orthotics marketplace with its innovative wearable foot supports designed to relieve pressure, improve alignment, and enhance balance for optimum comfort in any shoe.

Key Points: 
  • Female-owned StandStrong is shaking up the orthotics marketplace with its innovative wearable foot supports designed to relieve pressure, improve alignment, and enhance balance for optimum comfort in any shoe.
  • Unlike conventional orthotics, StandStrong does more than just support the arch or cushion the foot.
  • Made from a proprietary gel compound, StandStrong foot supports can be worn with any shoe style to accommodate a range of uses and activities without the hassle of clumsy inserts or arch supports.
  • The lower fin supports the cuboid bone, the keystone of the arch, to maximize overall stability, minimize pressure, and bolster proper foot alignment.