Muscle weakness

Fulcrum Therapeutics Announces Publication of Results from Phase 2b Clinical Trial of Losmapimod in Facioscapulohumeral muscular dystrophy (ReDUX4) in The Lancet Neurology

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Mercoledì, Maggio 8, 2024
Losmapimod, Fulcrum, Abdomen, NDA, The Lancet, Muscle weakness, REACH, Lancet Neurology, Disability, DUX4, Disease, Patient, FSHD, Face, Gene expression, Clinical trial, Therapy, File, Atrophy

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the publication of results from its Phase 2b clinical trial of losmapimod for the treatment of facioscapulopumeral muscular dystrophy (FSHD). The data are published in the peer-reviewed journal The Lancet Neurology.

Key Points: 
  • The data are published in the peer-reviewed journal The Lancet Neurology .
  • No serious adverse events related to the drug were reported, and there were no discontinuations of treatment due to adverse events.
  • In September 2023, Fulcrum announced the enrollment completion for the Phase 3 clinical trial evaluating losmapimod in patients with FSHD at sites in the United States, Canada, and Europe.
  • The clinical trial remains on track with topline data expected in Q4 2024.

Myasthenia Gravis Foundation of America (MGFA) Kicks off Annual National MG Patient Conference With Program Announcements

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Lunedì, Aprile 29, 2024
Online, Education, FDA, Patient, Language, Learning, Social work, Muscle weakness, Myasthenia gravis, Conference, Convention center, Myasthenia, Research, Human, Neurology, Disease, Registry, Fatigue, Caregiver, Community, Quality of life, MGFA, Nursing

TAMPA BAY, Fla., April 29, 2024 /PRNewswire/ -- More than 400 members of the myasthenia gravis (MG) rare disease community will come together this week at the MGFA National Patient Conference for support and a better understanding of how to manage their disease while learning about the latest in treatments and clinical studies.  

Key Points: 
  • Myasthenia Gravis Foundation of America (MGFA™) , the largest, leading patient advocacy organization solely dedicated to the myasthenia gravis community kicked off its annual conference today with a volunteer awards dinner and new program announcements.
  • The conference features patient stories and individuals diagnosed with myasthenia gravis as well as presentations and discussions from patients, caregivers, researchers, MG expert clinicians, and pharmaceutical and industry partners.
  • Bringing together MG patients and other members of the community to learn from and support each other is so powerful," said Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America.
  • MGFA Global MG patient Registry – The MGFA Global MG Patient Registry gives the MG community a loud voice in ensuring that research studies and clinical trials are built to evaluate the most promising MG research.

Revolutionary Scientists Honored for Advancements in Gene Therapy for Neuromuscular Diseases and RNA Discoveries: King Faisal Prize Laureates in Medicine, Professor Jerry Mendell, and in Science, Professor Howard Chang, Awarded

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Lunedì, Aprile 22, 2024
Science (journal), Mortality, FDA, Pediatric Research, Protein, Chromatin, Cell, Limb–girdle muscular dystrophy, Research, American Philosophical Society, National Cancer Institute, Molecular biology, Life, Gene, Quran, Food, LGMD, SMA, RNA, DMD, Spinal muscular atrophy, Infant, Dystrophin, Nationwide, Duchenne muscular dystrophy, Ageing, National academy, Hospital, Cancer research, Literature, DNA, Therapy, Gene expression, Muscular dystrophy, Chromosome, USD, Stanford University, Cancer, Islamic studies, Muscle weakness, Disease, Vilcek Foundation, Patient, Assay, Society, Listeria monocytogenes non-coding RNA, Pharmaceutical industry

Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.

Key Points: 
  • Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.
  • Gene therapy offers a solution by addressing this genetic anomaly, allowing the body to produce dystrophin and halt muscle degeneration.
  • Ludwig Professor of Cancer Research at Stanford University, has been awarded King Faisal Prize for Science in Biology.
  • Since 1979, King Faisal Prize in its 5 different categories has awarded 295 laureates who have made distinguished contributions to different sciences and causes.

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

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Lunedì, Aprile 15, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, TNNI2, Neuromuscular disease, University, Safety, Pharmacokinetics, MDA, Muscle weakness, NSAA, Duchenne, Patient, Muscular dystrophy, University of Florida, Multimedia, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.
  • ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
  • Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy (Duchenne).
  • ET to discuss the ARCH two-year data, and will be joined by Dr. Byrne, who will share his perspective of sevasemten and Becker.

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Retrieved on: 
Giovedì, Aprile 18, 2024
Eurostat, Diagnosis, Civil service commission, Mitochondrial encephalomyopathy, Headache, DSM-IV codes, Lactic acidosis, Weakness, MELAS, Cell, Disease, Seizure, Paratriathlon, Patient, Committee, Knowledge, Mitochondrion, Regulation, EMA, IRIS, European, COMP, Sponsor, European Commission, Safety, Muscle weakness, Pain, Vomiting, Marketing, Movement, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Phathom Pharmaceuticals Launches Direct-to-Consumer Campaign, “VOQUEZNA Can Kick Some Acid”

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Martedì, Marzo 26, 2024
Connected, HIV-1, Amazon Prime Video, Breast milk, Face, Hypoesthesia, Patient, Chronic pain, Clostridioides, Heartburn, Lymph, Magnesium, Diarrhea, Myalgia, Spine, Allergy, Muscle weakness, Vonoprazan, Stomach, Vitamin, Dizziness, Potassium, Hip, Risk, Tongue, Chills, DTC, Safety, Food, PPI, Rash, Food and Drug Administration, GERD, Enzyme inhibitor, Skin, Esophagitis, HIV, Rilpivirine, Kidney, FDA, Esophagus, B-12, Weakness, Infection, Indigestion, Smart TV, Seizure, Inflammation, Polyp, Calcium, Extrapyramidal system, Bone fracture, Nausea, Spasm, Urine, Fever, Clostridioides difficile, Bleeding, Blood, Wrist, Blister, Vitamin B12, Human, Medicine, Pharmaceutical industry

FLORHAM PARK, N.J., March 26, 2024 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, today announces the launch of its new broadcast ad and full-scale, Direct-to-Consumer (DTC) campaign, “VOQUEZNA Can Kick Some Acid,” to raise awareness of its powerful first-in-class treatment and encourage people to speak to their doctor about this new treatment option. VOQUEZNA (vonoprazan) is indicated for the healing and maintenance of healing of all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and for the relief of related heartburn. VOQUEZNA represents the first major innovation to the U.S. Erosive GERD market in over 30 years.1

Key Points: 
  • The campaign will also be featured on consumer-facing platforms across Facebook, Instagram, waiting room TVs in doctor offices, and digital banner ads.
  • “Phathom is excited to launch our first campaign directly to, and for, the people whose lives we strive to improve every day.
  • To view a video of the “VOQUEZNA Can Kick Some Acid” commercial, click here.
  • Talk with your healthcare provider about the possibility of fundic gland polyps if you have been on VOQUEZNA for a long time.

STEALTH BIOTHERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME

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Lunedì, Aprile 8, 2024
Friends, New Drug Application, Elamipretide, Patient, Barth syndrome, NDA, Syndrome, Ultra, Quality of life, Safety, Muscle weakness, Stealth, Fatigue, FDA, Food, Medicine, Pharmaceutical industry

NEEDHAM, Mass., April 8, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has accepted for filing its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome. The NDA is supported by the positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020. 

Key Points: 
  • Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020.
  • The FDA indicated that it is currently planning to hold an advisory committee meeting to discuss the application.
  • The application was assigned a standard review designation, which the Company has asked FDA to reconsider.
  • Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

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Martedì, Aprile 9, 2024
Eurostat, Civil service commission, Wasting, Diagnosis, Atrophy, Mutation, B cell, Weakness, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Death, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, Safety, European Commission, Muscle weakness, Marketing, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

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Martedì, Aprile 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

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Martedì, Aprile 9, 2024
Eurostat, Civil service commission, Eating, Diagnosis, Azathioprine, Disease, Dermatomyositis, Risk, Rash, Inflammation, Patient, Committee, Knowledge, Tissue, Regulation, EMA, IRIS, Mouth, European, Skin, COMP, Heart, Sponsor, European Commission, Immune system, Safety, Muscle weakness, Skeletal muscle, Stomach, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

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Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn