Neuromuscular medicine

Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial

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월요일, 3월 4, 2024
Therapy, Division, PD, PRO, Natural history, Doctor of Philosophy, Myotonic dystrophy, MD, Friends, Neurology, Hope, Neuromuscular medicine, Conference, DM1, Facial muscles, Safety, Muscular Dystrophy Association, Biomarker, RNA, Webcast, Patient, Elbow, Family, Disease, QMT, Pediatrics, AOC, Publication, MDA, Volume Eight, Episcopal conference, Partnership, Pharmaceutical industry

SAN DIEGO, March 4, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.

Key Points: 
  • These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1.
  • Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.
  • Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.
  • ET to discuss new positive long-term del-desiran (AOC 1001) data from the MARINA-OLE™ trial in people living with DM1.

Avidity Biosciences to Present New AOC 1001 Long-term Efficacy and Safety Data from MARINA-OLE™ Trial in People Living with Myotonic Dystrophy Type 1 (DM1) at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

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목요일, 2월 15, 2024
Therapy, Division, Doctor of Philosophy, MD, Clinical trial, DMD, Functional, Neurology, Neuromuscular medicine, Conference, DM1, Safety, Muscular Dystrophy Association, RNA, Webcast, Myotonic dystrophy, Pediatrics, AOC, Publication, MDA, Volume Eight, Episcopal conference

SAN DIEGO, Feb. 15, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the company will be presenting multiple posters from all three clinical development programs in rare muscle diseases, including new AOC 1001 long-term efficacy and safety data from the MARINA open-label extension (MARINA-OLE™) trial in people living with myotonic dystrophy type 1 (DM1) at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida.

Key Points: 
  • ET for a live video webcast event, which will be available on the company's website.
  • The company is hosting Volume 8 of its investor and analyst event series on March 4, 2024, beginning at 8:00 a.m.
  • ET to discuss new AOC 1001 long-term efficacy and safety data from the MARINA-OLE™ trial in people living with DM1.
  • A replay of the webcast will be archived on Avidity's website following the event.

National Institutes of Health Awards $45.1 Million NINDS Grant for CNM-Au8® Study in ALS

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목요일, 10월 5, 2023
ACT, Congress, ALS, Neuromuscular medicine, Columbia University Vagelos College of Physicians and Surgeons, MBA, Food, Doctor of Philosophy, Biomarker, Division, Research, Medicine, National Institute of Neurological Disorders and Stroke, FAAN, Master of Science, Neurology, NINDS, Safety, Columbia University, Survival, SALT, Disease, Surgeon, FDA, Collection, Clinical trial, DSM-IV codes, Microsoft Access, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Bangladesh Technical Education Board, MHS, CNM, Physician, 21st Century Cures Act, EAP, Patient, NIH, Pharmaceutical industry, MD, Stroke

In addition to this new EAP, Clene will continue to conduct its currently ongoing ALS EAP programs that have enrolled more than 200 participants since 2019.

Key Points: 
  • In addition to this new EAP, Clene will continue to conduct its currently ongoing ALS EAP programs that have enrolled more than 200 participants since 2019.
  • The EAP grant is part of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).
  • This EAP will provide eligible people living with ALS to work with their clinician to access CNM-Au8.
  • These data can help provide confirmatory support for the existing trial data Clene has gathered in its clinical trials.”
    This study is supported by NIH grant U01NS136023.

SOLVE FSHD Announces Appointment of Executive Director Dr. Eva Chin and Venture-Philanthropic Funding Structure

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수요일, 5월 11, 2022
Executive director, Pfizer, Investment, University, Specialization, Degenerative disease, Stanford University, GLOBE, Facioscapulohumeral muscular dystrophy, Hypertrophic cardiomyopathy, SOLVE, Myasthenia gravis, Solve, Doctor of Philosophy, Calcium, Industry, Leadership, MD, Muscle fatigue, Gene expression, SMA, Quality of life, History of the University of Maryland, College Park, Life, Goal, Cytokinetics, Research, ALS, Neuromuscular medicine, Pharmaceutical industry, Dietary supplement, Physiology, FSHD

VANCOUVER, British Columbia, May 11, 2022 (GLOBE NEWSWIRE) -- Following SOLVE FSHDs recent acclaimed launch, the organization is pleased to formally announce the appointment of Dr. Eva Chin as the Executive Director.

Key Points: 
  • VANCOUVER, British Columbia, May 11, 2022 (GLOBE NEWSWIRE) -- Following SOLVE FSHDs recent acclaimed launch, the organization is pleased to formally announce the appointment of Dr. Eva Chin as the Executive Director.
  • As the inaugural Executive Director for SOLVE FSHD, Dr. Chin will be responsible for aligning the organizations goals with the expertise of researchers, scientists and companies to promote and fund the discovery and development of new therapies for FSHD.
  • We are excited to gain her expertise and guidance to achieve the organizations goal of finding a cure for FSHD by December 2027, says Chip Wilson, SOLVE FSHD Founder.
  • Solve FSHD is funding innovative biotech and biopharma research and development activities that accelerate novel treatments of Facioscapulohumeral muscular dystrophy (FSHD) treatment.

Zogenix to Host Key Opinion Leader Webinar on Thymidine Kinase 2 Deficiency and MT-1621

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월요일, 11월 1, 2021
Myopathy, CDD, European Medicines Agency, Complutense University of Madrid, Natural history, Peaceful transition of power, Clinical trial, Columbia University, Albert Einstein College of Medicine, Publication, Research institute, Division, GLOBE, Degenerative disease, Research, Molecular biology, Children's Medical Research Institute, TK2, Epilepsy, KOL, Montreal Neurological Institute and Hospital, Columbia University Irving Medical Center, Pyrimidine, Lennox–Gastaut syndrome, Lucy, Continuation, Q&A, ALS, Seizure, DNA, Fenfluramine, Dravet syndrome, Neurological Institute of New York, NASDAQ, FDA, Neuromuscular medicine, Mitochondrial disease, Muscle, Pharmaceutical industry

EMERYVILLE, Calif., Nov. 01, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced that it will host a key opinion leader (KOL) webinar on thymidine kinase 2 deficiency (TK2d) and MT-1621 on Monday, November 8, 2021, at 12:00 PM Eastern Time.

Key Points: 
  • EMERYVILLE, Calif., Nov. 01, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced that it will host a key opinion leader (KOL) webinar on thymidine kinase 2 deficiency (TK2d) and MT-1621 on Monday, November 8, 2021, at 12:00 PM Eastern Time.
  • Dr. Galer will discuss the ongoing late-stage MT-1621 clinical program and remaining pathway to regulatory submissions.
  • She holds a doctorate from the Complutense University of Madrid with the thesis entitled: "Application of Personalized Medicine in Mitochondrial Pathology: treatment with pyrimidine nucleosides in thymidine kinase 2 deficiency (TK2d)".
  • The company has two additional late-stage development programs: one in a rare epilepsy called Lennox-Gastaut syndrome and one in a mitochondrial disease called TK2 deficiency.

Applied Therapeutics Reports Biomarker Data from Pilot Trial of AT-007 in SORD Deficiency

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월요일, 10월 25, 2021
Neurology, NEW, MD, Research, Peripheral nervous system, Diagnosis, University, Blood, Disease, Industry, Vii, Diabetic cardiomyopathy, CNS, Twitter, Patient, Ix, Private Securities Litigation Reform Act, Hereditary neuropathy with liability to pressure palsy, Applied psychology, Neuromuscular medicine, Xi, U.S. Securities and Exchange Commission, Disability, ID, Eye, Diabetic retinopathy, Government, Ribosomal s6 kinase, Sorbitol, Forward-looking statement, Patent, Intention, COVID-19, Lists of diseases, Investor relations, Metabolism, Nasdaq, ARI, Therapy, PMM2-CDG, Division, Motor neuron, Central nervous system, GLOBE, Doctor of Philosophy, Heart, Pharmaceutical industry

NEW YORK, Oct. 25, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported biomarker data from a pilot trial of AT-007 in patients with SORD Deficiency.

Key Points: 
  • Sorbitol Dehydrogenase Deficiency (SORD Deficiency) is a rare, progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons.
  • Patients with SORD Deficiency have 100 times higher sorbitol concentration in their blood compared with unaffected individuals.
  • In a pilot open-label study in 8 SORD Deficiency patients, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment.
  • Applied Therapeutics will host a conference call today, Monday, October 25, 2021, at 8:30 a.m. Eastern Time, to discuss data from a pilot trial of AT-007 in SORD deficiency.

PepGen Welcomes Esteemed Neurologist Dr. John Day to Scientific Advisory Board

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수요일, 10월 13, 2021
Degenerative disease, Myotonic dystrophy, Division, National Advisory Committee on Immunization, Organization, San Francisco, Professor, Duchenne muscular dystrophy, LinkedIn, Lists of diseases, DMD, Pathology, Coronavirus Scientific Advisory Board (Turkey), DM1, Neuromuscular medicine, Society, University of California, San Francisco, Albert Einstein College of Medicine, American Academy, GLOBE, Myotonic Dystrophy Foundation, University, Muscle, American Academy of Neurology, Twitter, Education, Edos, Pharmaceutical industry

BOSTON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- PepGen, Inc. , a company advancing next-generation oligonucleotide therapies for neuromuscular and neurologic diseases,today announcedthe appointment of neuromuscular neurologist John W. Day, M.D., Ph.D., to its Scientific Advisory Board.

Key Points: 
  • BOSTON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- PepGen, Inc. , a company advancing next-generation oligonucleotide therapies for neuromuscular and neurologic diseases,today announcedthe appointment of neuromuscular neurologist John W. Day, M.D., Ph.D., to its Scientific Advisory Board.
  • Dr. Day is a Professor of Neurology, Pediatrics (Genetics) and Pathology and Director of the Division of Neuromuscular Medicine at Stanford University.
  • Its my pleasure to welcome Dr. John Day, who brings decades of experience working and teaching in the neuromuscular disease space, to PepGens Scientific Advisory Board, said James McArthur, Ph.D., President and Chief Executive Officer of PepGen.
  • John is a renowned expert in neuromuscular diseases and particularly myotonic dystrophy, a severe, progressive disorder that currently has no cure.

Modis Therapeutics Announces Presentation at International World Muscle Society Congress 2018

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수요일, 10월 3, 2018
Health, Biotechnology, Nature, Biopharmaceutical, Pharmaceutical industry, Pharmacy, Therapy, Neuromuscular medicine, Neurology

OAKLAND, Calif., Oct. 3, 2018 /PRNewswire/ --Modis Therapeutics announced today that Dr. Michio Hirano, Professor of Neurology and Chief of the Division of Neuromuscular Medicine, Columbia University Irving Medical Center, has been invited to speak at the 23rd International Annual Congress of the World Muscle Society (WMS) which is being held October 2-6 in Mendoza, Argentina.

Key Points: 
  • OAKLAND, Calif., Oct. 3, 2018 /PRNewswire/ --Modis Therapeutics announced today that Dr. Michio Hirano, Professor of Neurology and Chief of the Division of Neuromuscular Medicine, Columbia University Irving Medical Center, has been invited to speak at the 23rd International Annual Congress of the World Muscle Society (WMS) which is being held October 2-6 in Mendoza, Argentina.
  • The presentation will highlight clinical results of treatment of TK2d patients with deoxynucleoside therapy.
  • Modis Therapeutics is working with Dr. Hirano, Columbia University Irving Medical Center and a consortium of international collaborators to develop deoxynucleoside combination therapy, MT1621, for TK2d.
  • 1,2
    Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with high unmet medical need.