Muscular dystrophy

Patient Organizations Gave $22 Billion in Grants for Research, Patient Services, and Access to Care Over the Last 5 Years, Says IQVIA Institute for Human Data Science

Retrieved on: 
Thursday, October 5, 2023
Health, Hospitals, Practice Management, General Health, Research, Science, Learning, The Michael J. Fox Foundation, Data science, Alzheimer's Drug Discovery Foundation, COVID-19, Muscular dystrophy, Food, Biomarker, Muscular Dystrophy Association, Drug development, Human, Organization, Therapy, Motion, Neurology, 21st Century Cures Act, European Medicines Agency, Disease, Patient, FDA, Education, Drug discovery, Rare, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Overalls, Registry, Pharmaceutical industry

Over the past five years, patient organizations have contributed $22.2 billion in grants to support patient services, research, and access to care.

Key Points: 
  • Over the past five years, patient organizations have contributed $22.2 billion in grants to support patient services, research, and access to care.
  • Nonprofit patient organizations have existed for more than 100 years and now number in excess of 3,000 in the United States.
  • The disease and mission focus of patient organizations has shifted and expanded over time as well.
  • As an example, more than 150 registries are now run by patient organizations, of which 62% focus on rare diseases, positioning patient organizations as custodians of data for their communities.

Atamyo Therapeutics Announces participation in Conferences and Key Scientific Communications on its LGMD programs

Retrieved on: 
Friday, September 29, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Muscular dystrophy, Natural history, International, Gene, C2, Rentschler, Part, ESGCT, Facial muscles, Neuromuscular disease, Diplomatic mission, Circulatory system, Rigshospitalet, Cell, Public, Oral, European Society of Clinical Microbiology and Infectious Diseases, Therapy, Conference, Pharmaceutical industry, Oyster farming, Nightclub, Rare-earth element, MD

Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation to forthcoming conferences and scientific communications on its programs targeting limb-girdle muscular dystrophies (LGMDs).

Key Points: 
  • Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation to forthcoming conferences and scientific communications on its programs targeting limb-girdle muscular dystrophies (LGMDs).
  • Stephane Degove, Atamyo CEO, will provide a corporate overview at two forthcoming conferences in October:
    Chardan's 7th Annual Genetic Medicines Conference, held in New York City:
    - Presentation on October 2, at 10:30 AM, in the Embassy room
    Cell & Gene Meeting on the Mesa, held in Carlsbad, CA, on October 10-12, 2023:
    - Presentation on October 10, at 5:15pm, at the Rentschler ATMP Ballroom
    Two upcoming communications at the 28th Annual Congress of the World Muscle Society (3-7 October 2023 in Charleston, SC)
    • Oral Presentation by A. Brureau (Genethon), at Ballroom C2 on October 4, 2023 from 18h15 to 18h45
    • Poster Board Number 282 at Ballroom C, on October 4, 2023, 17h15-18h15
    Natural history of limb girdle muscular dystrophy R9: one-year follow-up of a European cohort
    • Oral Presentation by J. Vissing (Copenhagen Neuromuscular Center, Rigshospitale) at Ballroom C2 on October 4, 2023 from 18h15 to 18h45
    • Poster Board Number 283 at Ballroom C, on October 4, 2023, 17h15-18h15
    Two communications describing Atamyo’s LGMD programs will be presented at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) that will take place on October 24-27, 2023 in Brussel (Belgium):
    • Oral presentation by Nicolai Preisler, MD, Rigshospitalet, Copenhagen, during the Cardiovascular and muscular diseases session, on Friday October 27, 11h30-13h00 – Le BEL, Tour & Taxis
    Oral presentation on Atamyo’s LGMD-R9 program at the 2023 International Limb-Girdle Muscular Dystrophy Conference (28-29 October 2023, Washington DC)
    • Oral presentation by John Vissing, MD, Rigshospitalet, Copenhagen, during the Clinical Trials Updates (Part 1) Session, on October 29, 11h00am-12h10pm – Constitution Ballroom, Grand Hyatt

Edgewise Therapeutics to Present on EDG-5506 for Becker Muscular Dystrophy at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Wednesday, September 27, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Dystrophinopathy, WMS, Muscular dystrophy, Becker muscular dystrophy, Drug, Trial of the century, ARCH, Congress, Edgewise, Natural history, Data, Therapy, Pharmaceutical industry, Becker

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present on EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, at the 28th International Annual Congress of the World Muscle Society (WMS).

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present on EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, at the 28th International Annual Congress of the World Muscle Society (WMS).
  • The conference will take place at the Charleston Convention Center in Charleston, South Carolina, from October 3-7, 2023.
  • Details of the Edgewise symposium and scientific posters at WMS:
    On Tuesday, October 3, 2023, at 4:30 pm ET, Edgewise will sponsor a symposium, “Becker Muscular Dystrophy Natural History and ARCH, an Open Label Study in Becker: Putting the Data in Context.” The symposium will feature presentations by Edgewise leadership and leading neuromuscular disease experts:
    Sam Collins, M.D., Ph.D., Vice President of Clinical Development, Edgewise Therapeutics
    Only registered conference attendees can register for the symposium.
  • Title: Characterization of short- and long-term proteomic response to the fast skeletal myosin inhibitor, EDG-5506, in Becker muscular dystrophy (P.254)
    The Edgewise symposium presentation and posters will be available on the Edgewise website when they are presented.

Edgewise Therapeutics Announces Initiation of GRAND CANYON, a Global Pivotal Study of EDG-5506 in Becker Muscular Dystrophy (Becker)

Retrieved on: 
Tuesday, September 26, 2023
Health, Physical Therapy, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Dystrophinopathy, Creatine kinase, Myopathy, Duchenne muscular dystrophy, Muscular dystrophy, Pharmacokinetics, Biomarker, Duchenne, NSAA, ARCH, PPMD, Entrepreneurship, Ageing, Therapy, Safety, Dietary supplement, Becker, Edgewise

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the start of enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker.

Key Points: 
  • Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the start of enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker.
  • EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy.
  • There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need.
  • GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker.

MilliporeSigma to Become First Provider to Offer Fully Integrated mRNA Services

Retrieved on: 
Tuesday, September 26, 2023
GMP, Sigma-Aldrich, Körber Process Solutions, Lipid, Cancer, Believer, Max, Merck Group, ECRC, Research, Experiment, Messenger, Molecular medicine, Helmholtz Association, Mab, Growth, Merck, Spacecraft, LNP, A-DNA, Muscular dystrophy, Vaccine, Pharmaceutical industry, BURLINGTON

The company today opened two new GMP-grade mRNA drug substance manufacturing sites in Darmstadt and Hamburg, Germany.

Key Points: 
  • The company today opened two new GMP-grade mRNA drug substance manufacturing sites in Darmstadt and Hamburg, Germany.
  • "We are now the first CTDMO to streamline the entire mRNA process for our clients."
  • MilliporeSigma also offers differentiated PCR-based technology for clinical and commercial mRNA manufacturing, providing clients with high-quality mRNA.
  • Its unique combination of mRNA expertise, technologies, regulatory knowledge, and product portfolios streamlines mRNA manufacturing and testing.

argenx Announces VYVGART (efgartigimod alfa) Authorized for Sale by Health Canada for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, September 21, 2023
Hair, University, Lancet Neurology, FC, Muscular dystrophy, GMG, Efgartigimod alfa, Netherlands, Sale, Muscular Dystrophy Canada, Euronext, Patient, University of Toronto, Division, ADAPT, Immunoglobulin G, SE, University Health Network, Acetylcholine receptor, Neonatal Fc receptor, Health Canada, The Lancet, Pharmaceutical industry, Neurology

With this regulatory milestone, VYVGART is the first-and-only neonatal Fc receptor (FcRn) blocker authorized for sale in Canada.

Key Points: 
  • With this regulatory milestone, VYVGART is the first-and-only neonatal Fc receptor (FcRn) blocker authorized for sale in Canada.
  • “There continues to be a significant unmet medical need for people living with gMG, who face debilitating muscle weakness and mobility issues.
  • The gMG community in Canada has long awaited new effective treatment advancements for this rare condition.
  • “Today is an important day for us as we deliver on our commitment to the gMG community to make VYVGART available to patients in Canada,” said John Haslam, General Manager argenx Canada.

Sarcomatrix Therapeutics Corp.’s Exclusive Research Partner Strykagen Awarded SBIR Phase 2 Grant from the National Institutes of Health (NIH) to Advance Small Molecules Promoting Muscle Regeneration

Retrieved on: 
Wednesday, September 20, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, MOA, Therapy, Office of the Director of Civil Aviation, SBIR, Muscular dystrophy, Small Business Innovation Research, Research, Bangladesh Technical Education Board, Sarcopenia, Investigational New Drug, Cachexia, Integrin, Corp, IND, NIH, Pharmaceutical industry

“We are grateful for the Phase 2 SBIR grant from the NIH for S-969, a continuation of our initial grant supporting the advancement of S-969 through early pre-clinical studies,” said Ryan Wuebbles, Ph.D., Chief Science Officer, Strykagen.

Key Points: 
  • “We are grateful for the Phase 2 SBIR grant from the NIH for S-969, a continuation of our initial grant supporting the advancement of S-969 through early pre-clinical studies,” said Ryan Wuebbles, Ph.D., Chief Science Officer, Strykagen.
  • “This grant allows us to evaluate the efficacy of S-969 in standard dose ranging models and complete IND enabling actions before entering the clinic.”
    Research reported in this publication was supported by the Office of The Director, National Institutes of Health of the National Institutes of Health under Award Number 2R44OD030543-04.
  • The content is solely the responsibility of the authors and does not represent the official views of the National Institutes of Health.
  • This press release contains forward-looking statements, which, aside from historical facts, include future projections subject to significant risks, especially concerning Sarcomatrix’ s development programs.

ATA-200, Atamyo Therapeutics’ Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program

Retrieved on: 
Tuesday, September 19, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Muscular dystrophy, Clinical trial, Pathology, Hope, CTA, Biomarker, AAV, Weakness, Patient, SGCG, Liver, Entrepreneurship, Therapy, Environment, Disease, DSM-IV codes, Safety, LGMD, Pharmaceutical industry, Vaccine, Dietary supplement, FKRP

No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo.

Key Points: 
  • No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo.
  • “ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy,” said Isabelle Richard, Ph.D., Co-founder and Chief Scientific Officer of Atamyo.
  • ATA-200, the gene therapy for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan.
  • In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein.

The FSHD Society's September 23 podcast gives voice to thousands fighting FSH muscular dystrophy

Retrieved on: 
Friday, September 22, 2023
Volunteering, Chicago Seven, Society, The Trial of the Chicago 7, Woman, Roll, Research, Central Time, FSHD, Walking, Facioscapulohumeral muscular dystrophy, Patient, NCIS, Criminal Minds, Very Good Bad Thing, Optum, City, Muscular dystrophy, Television, Disease, Pharmaceutical industry

RANDOLPH, Mass., Sept. 22, 2023 /PRNewswire-PRWeb/ -- The FSHD Society announced that this Saturday, September 23, it is holding its annual livestream celebration of the International Walk & Roll to Cure FSHD. In the podcast, FSHD Radio host Tim Hollenback will interview celebrities, scientists, and patient advocacy leaders who are lending their support to the Walk & Roll – the largest and only international campaign focused solely on raising funds and awareness for facioscapulohumeral muscular dystrophy (FSHD). This debilitating and rare form of muscular dystrophy affects nearly one million men, women, and children around the world.

Key Points: 
  • This debilitating and rare form of muscular dystrophy affects nearly one million men, women, and children around the world.
  • People across North America are taking part in the Society's sixth annual Walk & Roll to Cure FSHD, the organization's signature event.
  • Supporters across North America can register for and donate to the FSHD Society's Walk & Roll event by visiting FSHDSociety.org/Walk-Roll .
  • Everyone is invited to tune in to the FSHD Society Walk & Roll Facebook Live Celebration on September 23rd.

Coalition to Cure Calpain 3 in Partnership with the National Organization for Rare Disorders (NORD®) Launches LGMD2A/Calpainopathy Registry

Retrieved on: 
Thursday, September 21, 2023
Partnership, Drug development, Therapy, NORD, Parent, Community Participation, Policy, Research, National Organization for Rare Disorders, NIH Intramural Research Program, Muscle weakness, C3, Guardians of the Galaxy, Wasting, Patient, Health, Muscular dystrophy, Disease, Medical device, Nursing

Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials.

Key Points: 
  • Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials.
  • While the original registry served primarily to contact Calpainopathy patients, the new registry will have the capability to collect longitudinal data by having participants periodically update their information.
  • "The LGMD2A/Calpainopathy Registry will provide a complete picture of each patient's experience with this disease," shares Dr. Jennifer Levy, C3 Scientific Director.
  • "This new study has tremendous promise as a strong partnership that engages the patient community and addresses current knowledge gaps for Calpainopathy.