Myasthenia

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.

Dianthus Therapeutics Announces Initiation of Phase 2 MaGic Trial of DNTH103 In Generalized Myasthenia Gravis (gMG)

Retrieved on: 
Monday, February 26, 2024
Therapy, QMG, FDA, Dianthus, Food, Acetylcholine, Magic, Neurology, Safety, Myasthenia, University, GMG, Food and Drug Administration, Myasthenia gravis, Patient, Complement component 1s, Half-life, IND, Pharmaceutical industry

NEW YORK and WALTHAM, Mass., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Dianthus Therapeutics, Inc. (Nasdaq: DNTH), a clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics to treat severe autoimmune diseases, today announced the initiation of the Phase 2 MaGic trial of DNTH103 in patients with generalized Myasthenia Gravis. The initiation follows U.S. Food and Drug Administration (FDA) clearance of the Phase 2 Investigational New Drug (IND) application for DNTH103. Top-line results from this trial are anticipated in the second half of 2025.

Key Points: 
  • The initiation follows U.S. Food and Drug Administration (FDA) clearance of the Phase 2 Investigational New Drug (IND) application for DNTH103.
  • Top-line results from this trial are anticipated in the second half of 2025.
  • “Following our encouraging Phase 1 data demonstrating a 60-day half-life and potent, specific classical pathway inhibition, we are excited to rapidly advance DNTH103, our investigational active C1s inhibitor, into a Phase 2 study in generalized Myasthenia Gravis,” said Simrat Randhawa, M.D., Chief Medical Officer of Dianthus Therapeutics.
  • Secondary endpoints include Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) and Quantitative Myasthenia Gravis (QMG) score assessments.

UCB announces U.S. FDA approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized myasthenia gravis

Retrieved on: 
Tuesday, October 17, 2023
Neonatal Fc receptor, UCB, MuSK protein, Food, MG, University of North Carolina at Chapel Hill, MAC, System, Disease, Immunoglobulin G, Hospital, Plasmapheresis, Oklahoma Office of the Chief Medical Examiner, Brussels Stock Exchange, NMJ, GMG, Diarrhea, Physician, RAISE, U.S. FDA, Neuromuscular junction, University, Safety, Lancet Neurology, Patient, The Lancet, Week, MD, Allied health professions, Myasthenia, Quality of life, Muscle weakness, Acetylcholine receptor, Travel, QMG, Neuromuscular disease, Fatigue, FDA, Upper respiratory tract infection, Pathology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Priority review, ACHR, Weakness, Pharmaceutical industry, Vaccine, Medicine, Neurology, C5

It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.

Key Points: 
  • It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.
  • Patients were randomized in a 1:1 ratio to receive daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or placebo for 12 weeks.
  • The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.
  • The primary endpoint for the RAISE study was change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.

Cartesian Therapeutics Announces Landmark Study in The Lancet Neurology of First Successful Clinical Trial of RNA Cell Therapy in Autoimmunity

Retrieved on: 
Thursday, June 22, 2023
Research, Neurology, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Other Science, University, Therapy, Lancet Neurology, Phase II, Neurotoxicity, Efficacy, Immunoglobulin therapy, Clinical trial, Bone marrow, Oregon Health & Science University, NIH, Chapel Hill, DRS, University of South Florida, Myasthenia gravis, RNA, Autoimmunity, University of Miami, Bangladesh Technical Education Board, CAR, DSM-IV codes, National Institute of Neurological Disorders and Stroke, Cytokine release syndrome, MG, Patient, Physician, Northwestern Health Sciences University, Autotransplantation, NINDS, Paper, Toxic encephalopathy, American Neurological Association, Immunosuppression, Myasthenia, The Lancet, Lancet, Safety, Pharmaceutical industry, Vaccine, Stroke, Cartesian

Cartesian Therapeutics , a clinical-stage biotechnology company pioneering cell therapies for autoimmune diseases, announced today the publication of a landmark paper in The Lancet Neurology.

Key Points: 
  • Cartesian Therapeutics , a clinical-stage biotechnology company pioneering cell therapies for autoimmune diseases, announced today the publication of a landmark paper in The Lancet Neurology.
  • The study describes Descartes-08, a cutting-edge RNA CAR-T (rCAR-T) therapy administered to patients with generalized myasthenia gravis (MG), a debilitating autoimmune neurological disease.
  • This is the first clinical trial using rCAR-T to treat autoimmunity, and the first successful Phase 2 trial using an engineered cell therapy to treat autoimmunity.
  • Clinical benefit was sustained long-term for most patients, even months after completing the course of therapy.

'All United for MG' organises first-ever European Myasthenia Gravis Day on June 2nd leads to pan-European call for action supported by European leaders

Retrieved on: 
Thursday, June 1, 2023
AMIS, Evolis, United States corporate law, MBA, Doctor of Philosophy, MG, Patient, Frustration, Ageing, Health, Conditional sentence, Disease, Face, Policy, Health Economics, Weakness, Guilt, Leg, Approximation error, Sanders, SHE, MG3, Diagnosis, Rare, AMES, Caregiver, UOC, Employment, International, Depression, Eyelid, Neurology, Family, Arm, European Parliament, Anxiety, Quality of life, GP, Movement, Nursing, Pharmaceutical industry, Copper, Residential care, Myasthenia, F, MD, K, Let Us All Unite and Celebrate Together, EU, Myasthenia gravis, European, S, Qi

BRUSSELS, June 1, 2023 /PRNewswire/ -- The #AllUnitedforMG coalition, with the institutional support of argenx, brings together European patient associations and representatives from Belgium, France, Germany, Greece, Italy, Romania, Poland and Spain and aims to create awareness and gain recognition of this life-threatening disease, strengthen patients' rights for a better quality of life and obtain better patient care. The disease affects between 56.000 and 100.000 people in Europe 4.

Key Points: 
  • "Raising awareness about myasthenia gravis is not just about spreading knowledge, it's about empowering those who are living with it.
  • Our aim is to enhance patients' quality of life and reduce their mental burden," conclude the founders of the European Myasthenia Gravis Day.
  • "The first European Awareness Day for Myasthenia Gravis marks an important milestone towards increasing awareness and understanding of this rare disease.
  • Therefore we are very happy that there is finally a European Myasthenia Gravis Day to put a spotlight on this disease."

'All United for MG' organises first-ever European Myasthenia Gravis Day on June 2nd leads to pan-European call for action supported by European leaders

Retrieved on: 
Thursday, June 1, 2023
AMIS, Evolis, United States corporate law, MBA, Doctor of Philosophy, MG, Patient, Frustration, Ageing, Health, Conditional sentence, Disease, Face, Policy, Health Economics, Weakness, Guilt, Leg, Approximation error, Sanders, SHE, MG3, Diagnosis, Rare, AMES, Caregiver, UOC, Employment, International, Depression, Eyelid, Neurology, Family, Arm, European Parliament, Anxiety, Quality of life, GP, Movement, Nursing, Pharmaceutical industry, Copper, Residential care, Myasthenia, F, MD, K, Let Us All Unite and Celebrate Together, EU, Myasthenia gravis, European, S, Qi

BRUSSELS, June 1, 2023 /PRNewswire/ -- The #AllUnitedforMG coalition, with the institutional support of argenx, brings together European patient associations and representatives from Belgium, France, Germany, Greece, Italy, Romania, Poland and Spain and aims to create awareness and gain recognition of this life-threatening disease, strengthen patients' rights for a better quality of life and obtain better patient care. The disease affects between 56.000 and 100.000 people in Europe 4.

Key Points: 
  • "Raising awareness about myasthenia gravis is not just about spreading knowledge, it's about empowering those who are living with it.
  • Our aim is to enhance patients' quality of life and reduce their mental burden," conclude the founders of the European Myasthenia Gravis Day.
  • "The first European Awareness Day for Myasthenia Gravis marks an important milestone towards increasing awareness and understanding of this rare disease.
  • Therefore we are very happy that there is finally a European Myasthenia Gravis Day to put a spotlight on this disease."

Myasthenia Gravis Foundation of America (MGFA) Announces Official Start of MG Awareness Month Around the World

Retrieved on: 
Thursday, June 1, 2023
Education, DARE, Quality of life, Hope, Research, MGFA, Muscle weakness, MG, Patient, FDA, Myasthenia, Caregiver, Fatigue, Disease, Pharmaceutical industry, Nursing

BOSTON, June 1, 2023 /PRNewswire/ -- The MG Community around the world is turning Awareness into Action in June to educate everyone about the challenges of the rare neuromuscular disease myasthenia gravis (MG).

Key Points: 
  • Global myasthenia gravis community taking action in June to spread awareness about rare disease and highlight debilitating challenges experienced by patients.
  • The Myasthenia Gravis Foundation of America (MGFA™) , the largest, leading patient advocacy organization solely dedicated to the myasthenia gravis community announced the official start of MG Awareness Month in June.
  • MGFA focuses on funding promising research discoveries for better treatments while providing impactful programs, guidance, and education to support members of the MG Community.
  • The organization also raises funds through its DARE to CARE program that supports MG research grant funding and educational materials or events creation.

Myasthenia Gravis Foundation of America (MGFA) Announces Alliance with Patients Rising to Offer MG Patient Advocacy and Support Services

Retrieved on: 
Monday, February 27, 2023
Hysterectomy Educational Resources and Services Foundation, Learning, Patient, Rising, Disease, Life, MG, MGFA, Myasthenia gravis, Caregiver, Education, Myasthenia, Nursing

BOSTON, Feb. 27, 2023 /PRNewswire/ -- Two patient-centric advocacy organizations have created an alliance to help patients with chronic disease navigate the health care system.

Key Points: 
  • MGFA is aligning with Patients Rising to offer Patient Helpline to help solve MG patient problems and provide services.
  • The Myasthenia Gravis Foundation of America (MGFA™) , the largest, leading patient advocacy organization solely dedicated to the myasthenia gravis (MG) community is aligning with Patients Rising , a Washington D.C.-based non-profit that provides education, resources, and advocacy for people living with chronic and life-threatening illnesses.
  • Patients Rising will offer tools and strategies to empower people with myasthenia gravis to obtain support and services as well as professional training programs focused on patient advocacy.
  • "We are excited to offer our patient support and education services to myasthenia patients, and we greatly value our alliance with the MGFA," said Jim Sliney Jr, Patients Rising's executive director.

UCB announces rozanolixizumab BLA for the treatment of generalized myasthenia gravis filed with U.S. FDA and designated for Priority Review

Retrieved on: 
Friday, January 6, 2023
Steroid, Priority review, CHMP, Protein, Food, Muscle, FC, Neonatal Fc receptor, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MG, BLA, Royal commission, Myasthenia, Eyelid, UCB, Acetylcholine receptor, Muscle weakness, SC, European Directive on Traditional Herbal Medicinal Products, Disease, Patient, Degenerative disease, Medicine, Committee, Committee for Medicinal Products for Human Use, Safety, European Commission, Neuromuscular junction, Marketing, FDA, Biologics license application, MAA, GMG, EMA, MuSK protein, Diagnosis, Prevalence, Priority, Pharmaceutical industry, Vaccine, Agency

Validation confirms that the application is complete and the formal review process by the EMA's Committee for Medicinal Products for Human Use (CHMP) can begin.

Key Points: 
  • Validation confirms that the application is complete and the formal review process by the EMA's Committee for Medicinal Products for Human Use (CHMP) can begin.
  • Orphan designation was granted by the European Commission in April 2020 to rozanolixizumab for the treatment of myasthenia gravis.5
    UCB expects to receive feedback from both the FDA and EMA during the second quarter of 2023.
  • We look forward to working with the FDA and EMA to help bring this new treatment option to patients."
  • UCB is currently investigating two potential therapies with different modes of action for the treatment of gMG.