Charcot–Marie–Tooth disease

InFlectis BioScience Receives Approval from French Regulatory Authority to Conduct a Phase 2 Clinical Trial for IFB-088 (Icerguastat) for the Treatment of Amyotrophic Lateral Sclerosis

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星期一, 一月 10, 2022
Safety, Amyotrophic lateral sclerosis, Charcot–Marie–Tooth disease, Amyotrophy, Disease, Protein, ANSM, Patient, Degenerative disease, ALS, Integrated stress response, BioScience, CMT, SAS, Therapy, ISR, Riluzole, Pharmaceutical industry, Telescopic sight

Nantes, France--(Newsfile Corp. - January 10, 2022) - InFlectis BioScience SAS, a clinical-stage company developing innovative therapeutics for neuromuscular diseases by harnessing the Integrated Stress Response (ISR), receives approval from the French Regulatory Authority ANSM to start a Phase 2 clinical trial evaluating IFB-088 for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • Nantes, France--(Newsfile Corp. - January 10, 2022) - InFlectis BioScience SAS, a clinical-stage company developing innovative therapeutics for neuromuscular diseases by harnessing the Integrated Stress Response (ISR), receives approval from the French Regulatory Authority ANSM to start a Phase 2 clinical trial evaluating IFB-088 for the treatment of Amyotrophic Lateral Sclerosis (ALS).
  • The study is a double-blind, placebo-controlled, exploratory randomized clinical trial to assess the safety and efficacy of IFB-088.
  • InFlectis BioScience is a France-based clinical stage company developing first-in-class therapies for neuromuscular diseases.
  • InFlectis drug candidates keep this command center operating to prevent downstream effects that lead to disease.

Experienced Pharmaceutical Industry Executive Dr. Elisabeth Svanberg Appointed as Chairman of Pharnext Board of Directors

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星期四, 十二月 23, 2021
Intention, Charcot–Marie–Tooth disease, Regulatory News Service, Drug development, University of Gothenburg, Peripheral nervous system, Doctor of Philosophy, Meanings of minor planet names: 8001–9000, Patient, Drug discovery, Time, Neurodegeneration, Alzheimer's disease, Prospectus, Bristol Myers Squibb, Industry, University, ISIN, AMF, Metabolism, Artificial intelligence, Therapy, Board, Director, BMS, Janssen Pharmaceuticals, Mission statement, Associate, Pharmaceutical industry, Genomics, Trial of the century, URD, Intelligence, MD, Company, Gothenburg, Neurological disorder, CET, Type 2 diabetes, Management, Security (finance), Serono

Experienced Pharmaceutical Industry Executive Dr. Elisabeth Svanberg Appointed as Chairman of Pharnext Board of Directors

Key Points: 
  • Experienced Pharmaceutical Industry Executive Dr. Elisabeth Svanberg Appointed as Chairman of Pharnext Board of Directors
    Dissemination of a French Regulatory News, transmitted by EQS Group.
  • Experienced Pharmaceutical Industry Executive Dr. Elisabeth Svanberg Appointed as Chairman of Pharnext Board of Directors
    PARIS, France, December 23rd, 2021, 8:30 am 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 today the appointment of Dr. Elisabeth Svanberg MD, PhD to succeed Michel de Rosen as Chairman of the Board of Directors effective January 1st, 2022.
  • Michel de Rosen has served as Chairman of the Board since June 2016 and will continue to serve as a Non-Executive Director.
  • She is an experienced non-executive director serving on the Board of Galapagos NV, Egetis Pharmaceuticals, Amolyt and Swedish Orphan Biovitrum (SOBI).

Pharnext appoints Dr. Burkhard Blank as new Chief Medical Officer and Head of Research & Development

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星期一, 十一月 22, 2021
Regulatory News Service, Intelligence, Genomics, Neurological disorder, Industry, Company, Research and development, Mission statement, Peripheral nervous system, Acorda Therapeutics, Therapy, Neurodegeneration, Alzheimer's disease, Prospectus, NDAS, Boehringer Ingelheim, Pharmacovigilance, CET, Parkinson's disease, Phase, EMA, Artificial intelligence, ISIN, Chief medical informatics officer, FDA, Internal medicine, Burchard, Drug discovery, Intention, AMF, Medicine, Life, Charcot–Marie–Tooth disease, CMO, URD, Security (finance), Medical device, Pharmaceutical industry

Dr. Burkhard Blank is an experienced leader in global drug development, medical and regulatory affairs, and pharmacovigilance with more than 25 years of industry experience.

Key Points: 
  • Dr. Burkhard Blank is an experienced leader in global drug development, medical and regulatory affairs, and pharmacovigilance with more than 25 years of industry experience.
  • He joins Pharnext from Acorda Therapeutics where he held the position of CMO and Head of Research & Development for the past six years.
  • Prior to this, Dr Blank served as CMO for several biopharmaceutical companies, including Boehringer Ingelheim, Herantis Pharma, and Mersana Therapeutics.
  • Dr. David Horn Solomon, Chief Executive Officer of Pharnext, commented: "I am delighted to welcome Burkhard to the team and look forward to working with him.

Passage Bio Reports Third Quarter 2021 Financial Results and Provides Recent Business Highlights

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星期四, 十一月 4, 2021
Safety, COVID-19, Central nervous system disease, Research, Charcot–Marie–Tooth disease, University, Metachromasia, Failure, CNS, Data, Amyotrophy, Security (finance), U.S. Securities and Exchange Commission, Patient, Krabbe disease, Investor, Private Securities Litigation Reform Act, Infant, SEC, PASG, Frontotemporal dementia, Risk, Adult, Research and development, Company, Beta-galactosidase, Clinical trial, Webcast, GM1, Temporal lobe epilepsy, IND, Program, R, Nasdaq, Serum, Cerebrospinal fluid, Amyotrophic lateral sclerosis, Metachromatic leukodystrophy, GLOBE, Administration, Pharmaceutical industry

ET

Key Points: 
  • ET
    PHILADELPHIA, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today reported financial results for the third quarter ended September 30, 2021 and provided recent business highlights.
  • As we close this year, we remain steadfastly focused on strong execution of our three clinical programs.
  • Appointed Maxine Gowen, Ph.D., as chairwoman of the board of directors in August 2021: This appointment followed the unexpected passing of Passage Bio co-founder and former board chair Dr. Tadataka Yamada.
  • General and Administrative (G&A) Expenses:G&A expenses were $15.0 million for the third quarter ended September 30, 2021, compared to $7.8 million for the same quarter in 2020.

Pharnext strengthens its management team with three key appointments to support ongoing development of PXT3003 toward approval and commercialization

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星期二, 十月 19, 2021
ISIN, Gastroenterology, Patient, Senior Consultant, Pharmaceutics, Neurodegeneration, URD, The Company, IIT BHU, Alexion Pharmaceuticals, Neurology, Drug discovery, Intelligence, Xavier University, Intention, Charcot–Marie–Tooth disease, AMF, Tablet, Mission statement, Artificial intelligence, Abhijit, CMC, HEC Paris, General manager, Genetics, Marketing, Regulatory News Service, Growth, RAC, Alzheimer's disease, Genomics, Certification, Master, Prospectus, Trial of the century, GlaxoSmithKline, Regulatory affairs, Raj Thota, CET, Villanova University, Degenerative disease, COO, IIT, Therapy, Company, Security (finance), Pharmaceutical industry

in Pharmaceutical Technology from IIT BHU, India, and M.B.A. in general management from Xavier University USA.

Key Points: 
  • in Pharmaceutical Technology from IIT BHU, India, and M.B.A. in general management from Xavier University USA.
  • He also completed a Regulatory Affairs Certification (RAC) from Regulatory Affairs Professional Society and Lean Six Sigma Greenbelt Certification from Villanova University, USA.
  • Raj's extensive experience in pharmaceutical development and Abhijit's past work in navigating drug development with global regulatory authorities will considerably augment the strength of our senior management team.
  • Our pivotal Phase III clinical study of PXT3003 in CMT1A, the PREMIER trial, is currently ongoing in the US and Europe and remains our key focus."

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

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星期一, 十月 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).

HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for Charcot-Marie-Tooth (CMT)

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星期二, 十月 12, 2021
Nerve Damage, Drug discovery, Program, Chronic pain, Neuron, Entrepreneurship, FDA, Charcot–Marie–Tooth disease, HNF, CEO, Founder, Muscle atrophy, Partnership, Patient, Disease, Research, CMT, Pharmaceutical industry

Rarebase is a public benefit biotechcompany focused on accelerating therapy development for rare diseases with its tech-enabled drug discovery platform called "Function".

Key Points: 
  • Rarebase is a public benefit biotechcompany focused on accelerating therapy development for rare diseases with its tech-enabled drug discovery platform called "Function".
  • Rarebase will screen a compound library of thousands of FDA-approved drugs and novel drugs, targeting ten subtypes of Charcot-Marie-Tooth (CMT).
  • If such treatments are out there for CMT, we will find them," said Onno Faber, Co-Founder and CEO of Rarebase.
  • We believe the Rarebase partnership will enable FDA approved drugs for clinical use for CMT.

InFlectis BioScience Expands Leadership Team with Three Experienced International Appointments: Mark Pykett Is Appointed Chairman, Béatrice Lejeune Is Appointed Chief Regulatory Officer, and Prof. Brian Popko Joins Scientific Advisory Board

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星期一, 十月 4, 2021
CNS, Atomic Energy Regulatory Board, Integrated stress response, Therapy, Amyotrophic lateral sclerosis, SAS, Coronavirus Scientific Advisory Board (Turkey), Charcot–Marie–Tooth disease, Board, Popular Executive Committee of Valencia, Executive Committee, ISR, Disease, Degenerative disease, ALS, CMT, Research, BioScience, Protein, Pharmaceutical industry, Telescopic sight

Dr. Mark Pykett, as the chairman, brings more than 20 years of experience managing and growing development-stage biopharmaceutical companies.

Key Points: 
  • Dr. Mark Pykett, as the chairman, brings more than 20 years of experience managing and growing development-stage biopharmaceutical companies.
  • Industrial pharmacist, Batrice Lejeune, serving in the newly-created role of Chief Regulatory Officer, will contribute with her expertise in bringing drugs to market.
  • Prof. Brian Popko joins InFlectis as a scientific advisory board member with more than 30 years of research experience in the areas of CNS disease.
  • InFlectis' drug candidates keep this command center operating to prevent downstream effects that lead to disease.

Patient-Powered Registry Boosts the Study of Charcot-Marie-Tooth Disease and Collects Data for Clinician Observation

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星期五, 九月 3, 2021
Patient, IRB, CMT, Awareness, Government, Observation, Drug discovery, AI, Charcot–Marie–Tooth disease, Quality of life, Drug development, Peripheral neuropathy, HNF, Diagnosis, IPhone, Research, Industry, HIPAA, Goal, Acupuncture & Electro-Therapeutics Research, Gaps, Disease, Data collection, Pharmaceutical industry

HNF's Global Registry for Inherited Neuropathies (GRIN), an IRB-approved, online database collects genetic reports, and specific diagnostic and medical tests, as well as self-reported patient data through the use of surveys.

Key Points: 
  • HNF's Global Registry for Inherited Neuropathies (GRIN), an IRB-approved, online database collects genetic reports, and specific diagnostic and medical tests, as well as self-reported patient data through the use of surveys.
  • Additionally, visual documentation of challenges with activities of daily living, and expert assessment of the progression of the disease is collected.
  • Natural history studies are critical to understanding conditions such as Charcot-Marie-Tooth (CMT) and advancing research to discover and develop new therapies.
  • HNF identified several gaps in existing CMT natural history studies: the lack of collaboration amongst stakeholders, the lack of clinician observation of clinically relevant data, and the need to enroll more CMT patients.

20 years later, the Hereditary Neuropathy Foundation brings back its "eye-catching" PSA in support of Charcot-Marie-Tooth Awareness Month

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星期三, 九月 1, 2021
Industry, Nerve Damage, Friends, Quality of life, Muscle atrophy, Peripheral neuropathy, Gene, Charcot–Marie–Tooth disease, Public service, Diagnosis, Family, Social media, CMT, Research, Government, Drug discovery, PSA, Disease, HNF, Awareness, Injury, Acupuncture & Electro-Therapeutics Research, Multimedia, Bullying, Public service announcement, The, Motion, Chronic pain, Patient, Self-care, Atrophy, CEO, Movement, Physical education, Facebook, Muscle weakness, View, Drug development, Pharmaceutical industry, Nightclub, Online shopping

NEW YORK, Sept. 1, 2021 /PRNewswire/ -- The Hereditary Neuropathy Foundation (HNF), an advocacy and research non-profit 501(c)3 organization, today brings back its "eye-catching" Public Service Announcement (PSA) 20 years after it was initially launched.

Key Points: 
  • NEW YORK, Sept. 1, 2021 /PRNewswire/ -- The Hereditary Neuropathy Foundation (HNF), an advocacy and research non-profit 501(c)3 organization, today brings back its "eye-catching" Public Service Announcement (PSA) 20 years after it was initially launched.
  • Currently, thousands of people with CMT are undiagnosed, yet are suffering with the symptoms of this chronic and often disabling disease.
  • Children are often pushed into Physical Education class and other activities that are not adapted to their special needs.
  • Show your support for CMT families and increase awareness across the globe by updating your Facebook profile photo with your exclusive frame.