Muscular dystrophy

Positive Results from Genethon’s Gene Therapy Trial for Duchenne Muscular Dystrophy, Advances of Its Spinoff Atamyo in the LGMD and Cardiomyopathy Fields, Highlighted in Latest Newsletter

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måndag, juni 3, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Newsletter, Interview, Mass, Data monitoring committee, Publication, Therapy, Muscular dystrophy, Duchenne muscular dystrophy, Clinical trial, Gene, Muscular Dystrophy Association, European

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.

Key Points: 
  • Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.
  • Also in an interview, Roger Hajjar, M.D., Director of the Gene and Cell Therapy Institute of Mass General Brigham in Boston, renowned gene therapy researcher and entrepreneur, offered insights on what attracted him to join Atamyo Therapeutics as the newest board member of the Genethon spinout company.
  • “What resonated with me was the fact that Atamyo is a research endeavor by investigators within Genethon whose work is being translated into the clinic,” Dr. Hajjar said.
  • It’s definitely a new model.”
    Genethon CEO Frederic Revah, Ph.D., commenting on gene therapy trials for Duchenne Muscular Dystrophy, observed, “The effective dose in this first phase of the trial is lower than that used in other gene therapy programs using microdystrophin.

Hope Biosciences Research Foundation Announces Topline Results of Cell Therapy Clinical Trial in Long COVID

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fredag, maj 31, 2024
Stem Cells, Biotechnology, FDA, Health, Philanthropy, General Health, Research, Foundation, Science, COVID-19, Clinical Trials, Patient, Lupus, Visual analogue scale, Ataxia, Cerebral palsy, Muscular dystrophy, Palliative care, Doctor of Philosophy, Stroke, ALS, Placebo, Protocol, Multiple sclerosis, Primary lateral sclerosis, Pancreatic cancer, Hemispherectomy, Polyneuropathy, COVID, Fatigue, B cell, PLS, Central nervous system, Pharmaceutical industry

Houston-area clinical research organization Hope Biosciences Research Foundation (HBRF) today shares topline results of a randomized, placebo-controlled Phase II study ( NCT05126563 ) to evaluate Hope Biosciences’ adipose‑derived allogeneic mesenchymal stem cell therapy (HB-adMSCs) for patients with Post‑COVID‑19 syndrome.

Key Points: 
  • Houston-area clinical research organization Hope Biosciences Research Foundation (HBRF) today shares topline results of a randomized, placebo-controlled Phase II study ( NCT05126563 ) to evaluate Hope Biosciences’ adipose‑derived allogeneic mesenchymal stem cell therapy (HB-adMSCs) for patients with Post‑COVID‑19 syndrome.
  • The trial enrolled 79 participants, with 39 subjects in the treatment group and 40 in the placebo group; 34 participants completed the study from the treatment group, and 30 from the placebo group.
  • The 26-week study mandated four infusions of 200 million stem cells, for a total of 800 million cells.
  • The study announced in this release is made possible in part due to support from The John S. Dunn Foundation.

Satellos Announces Formation of Clinical Advisory Board to Support Advancing SAT-3247 in Clinical Trial Development for Duchenne Muscular Dystrophy

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tisdag, maj 28, 2024
Science, Biotechnology, Research, Pharmaceutical, Managed Care, Health, Genetics, Clinical Trials, Patient, FSHD, Hospital for Sick Children, UCLA, MSCL, Tic disorder, FDA, Drug development, FAAN, Myotonic dystrophy, DMD, CAB, CMO, Shirin David, VCU, Professor, Hospital, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, CRISPR, Therapy, TSX, OTCQB, Muscular dystrophy, Pharmaceutical industry

“The formation of this Clinical Advisory Board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” said Frank Gleeson, Co-founder and CEO of Satellos.

Key Points: 
  • “The formation of this Clinical Advisory Board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” said Frank Gleeson, Co-founder and CEO of Satellos.
  • Research in his laboratory focuses on utilizing genome editing technologies like CRISPR for the development of therapeutic approaches for neurogenetic disorders.
  • Francesco Muntoni, M.D., Director, Dubowitz Neuromuscular Centre, Great Ormond Street UCL Institute of Child Health, is focused on advancing novel therapeutics in translational and clinical research for pediatric neuromuscular disorders, especially Duchenne muscular dystrophy.
  • Dr. Shieh’s principal clinical interests include Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and myotonic dystrophy.

Muscular Dystrophy Association’s Impact to End ALS Continues with Fourth Annual Lou Gehrig Day at Major League Baseball Games

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tisdag, maj 21, 2024
Campaign, New York Yankees, Doctor of Philosophy, Discord, Neuromuscular disease, Play, Organization, Friends, Muscular Dystrophy Association, Major League Baseball, Disease, ALS, Society, Research, Yankee, Social media, San Francisco Giants, Volunteering, Q&A, MLB, Muscular dystrophy, MDA

Also known as Lou Gehrig’s disease ALS took the life of the famed Yankee slugger at the age of 37.

Key Points: 
  • Also known as Lou Gehrig’s disease ALS took the life of the famed Yankee slugger at the age of 37.
  • The ALS community and teams from MLB honor Lou Gehrig Day on June 2, marking the date when Gehrig became the Yankees' starting first baseman, and the same date he passed away from ALS in 1941.
  • MDA’s recognition of Lou Gehrig Day at 17 ballparks across the country raises awareness and funds for its mission in support of families living with ALS.
  • We commend Major League Baseball for their steadfast support of Lou Gehrig Day, empowering baseball enthusiasts to contribute to ALS breakthroughs," said Donald S. Wood, PhD, President and CEO, MDA.

MDA's Pinup & Roundup Campaigns Kick Off Across the Nation to Support Summer Camp for Kids and Young Adults Living with Neuromuscular Diseases

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måndag, maj 20, 2024
Magic, Neuromuscular disease, Hope, Alaska Commercial Company, Friends, Muscular dystrophy, Muscular Dystrophy Association, FDA, Ageing, Counselor, Life, Sport, Community engagement, Senior, Research, Points of the compass, Circle K, Vallarta Supermarkets, Volunteering, Summer camp, American Furniture Warehouse, Child, Swimming, Family, MDA, Cabin, Tourism

The funds raised go towards crucial support for MDA Summer Camp, offering transformative experiences for kids and young adults aged 8-17 who are living with muscular dystrophy or related neuromuscular diseases.

Key Points: 
  • The funds raised go towards crucial support for MDA Summer Camp, offering transformative experiences for kids and young adults aged 8-17 who are living with muscular dystrophy or related neuromuscular diseases.
  • Additionally, funds from this campaign go towards accelerating research, advancing care, and advocating for families living with neuromuscular diseases.
  • MDA Summer Camp has been offered to kids and young adults living with neuromuscular diseases since 1955.
  • Reflecting on the experience as a volunteer counselor at MDA Summer Camp, a camp counselor at the Illinois camp shared, “Camp is pure magic.

Muscular Dystrophy Association Celebrates Historic Advancements in Accessible Air Travel as Congress Passes FAA Reauthorization

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onsdag, maj 15, 2024
Uncertainty, Atmosphere of Earth, Wheelchair, Doctor of Philosophy, Air travel, Legislation, Rail transport by country, Disability, Muscular dystrophy, Muscular Dystrophy Association, Travel, Paralyzed Veterans of America, MDA, Dignity, Fear, Congress, Atmosphere, Hill, Senate, House, Public policy, Federal Aviation Administration, Pain, DOT

Washington, D.C., May 15, 2024 (GLOBE NEWSWIRE) -- Today, the Muscular Dystrophy Association (MDA) celebrates a landmark achievement in accessible air travel as Congress grants final passage to the Federal Aviation Administration (FAA) reauthorization bill - H.R.3935, the Securing Growth and Robust Leadership in American Aviation Act .

Key Points: 
  • Washington, D.C., May 15, 2024 (GLOBE NEWSWIRE) -- Today, the Muscular Dystrophy Association (MDA) celebrates a landmark achievement in accessible air travel as Congress grants final passage to the Federal Aviation Administration (FAA) reauthorization bill - H.R.3935, the Securing Growth and Robust Leadership in American Aviation Act .
  • "This passage is the most significant advancement in accessible air travel in nearly four decades.
  • “The Muscular Dystrophy Association’s Accessible Air Travel advocacy campaign channeled the neuromuscular and the entire disability communities’ efforts to make it quite clear that mistreating people and damaging wheelchairs during air travel is unacceptable.
  • “With the final passage of the FAA reauthorization bill, the entire community of families served by Muscular Dystrophy Association look forward to a future where air travel is truly accessible to all.”
    "Finally, with the new reforms included in the FAA Reauthorization, there's hope for a transformation in air travel for people with disabilities.

Dutch Bros to hold annual Drink One for Dane giveback

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måndag, maj 13, 2024
DNA, Neuromuscular disease, Hope, Heart, Muscular Dystrophy Association, Disease, Research, Face, MDA, Muscular dystrophy, Amusement park

GRANTS PASS, Ore., May 13, 2024 /PRNewswire/ -- Help Dutch Bros support the Muscular Dystrophy Association (MDA) and their mission to end ALS!

Key Points: 
  • GRANTS PASS, Ore., May 13, 2024 /PRNewswire/ -- Help Dutch Bros support the Muscular Dystrophy Association (MDA) and their mission to end ALS!
  • For every drink sold on Friday, May 17, the Dutch Bros Foundation will make a donation to MDA.
  • "When we lift our cups to Drink One for Dane, we're not only honoring the legacy of Dane Boersma, we're actually igniting hope across the ALS and neuromuscular disease communities.
  • Every year Dutch Bros holds Drink One for Dane as a way to support research to find a cure for the disease.

Kate Therapeutics Unveils Platform and Pipeline Progress at American Society of Gene & Cell Therapy 2024 Annual Meeting

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torsdag, maj 9, 2024
Mortality, FDA, University, Facioscapulohumeral muscular dystrophy, Mouse, RNA interference, Safety, Tissue, Heart, Animal, Death, MD, Primate, Cell, Duchenne, Science, Disease, Rockefeller University, Patient, Liver, Society, Gene, X-linked myotubular myopathy, Pediatrics, Gene expression, Judgement, Duchenne muscular dystrophy, Capsid, Annual general meeting, SAN, Muscular dystrophy, Vaccine

SAN DIEGO, May 9, 2024 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, will present detailed preclinical efficacy and safety results on its pipeline and platform at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting being held May 7-11, 2024, in Baltimore, MD and virtually. The results support the best-in-class potential of KateTx's newest generation of skeletal muscle- and heart-targeted, liver de-targeted engineered MyoAAV capsids and gene regulation technology to treat Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and other devastating genetic skeletal muscle and heart diseases.

Key Points: 
  • KateTx remains on track to select development candidates for additional muscle and cardiac programs in the near term.
  • "KateTx has made significant advances over the past 12 months," said Kevin Forrest, Ph.D., co-founder, president and CEO of KateTx.
  • KateTx's results show the potential to give a one-time gene therapy that potently suppresses DUX4 whenever it is expressed in skeletal muscles.
  • In vitro, the company's MyoAAV-LD mediated RNAi gene therapy candidate showed potent knockdown of DUX4 in FSHD patient myotubes with no off-target effects.

Pliant Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results

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måndag, maj 6, 2024
Idiopathic pulmonary fibrosis, FVC, Investment, Hospital, Safety, Primary sclerosing cholangitis, Research, IPF, Collagen, Trial of the century, PET, Acceleration, DMD, Tumor microenvironment, PSC, European, Novartis, Week, Therapy, Vital capacity, Muscular dystrophy, Patient, TGF, Fibrosis, Cirrhosis, Integrin, Duchenne muscular dystrophy, Biomarker, Ethics committee, Massachusetts General Hospital, ELF, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., May 06, 2024 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a late-stage clinical biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, today provided a corporate update and reported first quarter 2024 financial results.

Key Points: 
  • As part of the adaptive Phase 2b/3 implementation, the Phase 2b portion of BEACON-IPF was upsized from 267 patients to 360 patients.
  • Enrollment is progressing and we expect to complete enrollment of the upsized Phase 2b study expected in the first quarter of 2025.
  • The increase was due to higher operating expenses coupled with a decrease in collaboration revenues under the Novartis agreement during the quarter.
  • As of March 31, 2024, the Company had cash, cash equivalents and short-term investments of $483.9 million.

Edgewise Therapeutics Reports First Quarter 2024 Financial Results and Recent Business Highlights

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torsdag, maj 9, 2024
Health, Communications, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Public Relations, Investor Relations, TNNI2, Safety, Pharmacokinetics, DUNE, Duchenne, McArdle, Ageing, Exercise, Creatine kinase, Patient, MRI, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Muscular dystrophy

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.
  • The Company expects to report CANYON data in the fourth quarter of 2024.
  • Research and development (R&D) expenses were $27.7 million for both the first quarter of 2024 and for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $7.1 million for the first quarter of 2024, compared to $6.2 million for the immediately preceding quarter.