Muscular Dystrophy Association

Burn Boot Camp and Muscular Dystrophy Association Team Up for 8th Annual 'Be Their Muscle' Philanthropic Event

Retrieved on: 
Monday, March 25, 2024
Research, Employment, Entrepreneurship, Skeletal muscle, MDA, ALS, Partnership, Air travel, Education, Duke University Hospital, Social media, Muscular dystrophy, Muscular Dystrophy Association, Senior, Empowerment, Duchenne muscular dystrophy, Duchenne, Clinical trial, FDA, Summer camp, Pharmaceutical industry

Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.

Key Points: 
  • Charlotte, North Carolina, March 25, 2024 (GLOBE NEWSWIRE) -- Burn Boot Camp, a leading boutique fitness franchise, has teamed up with the Muscular Dystrophy Association (MDA) for its eighth annual national 'Be Their Muscle' philanthropic event.
  • Muscular Dystrophy Association is one of Burn Boot Camp's longest-standing national philanthropic partners," explained Morgan Kline, CEO and Co-Founder of Burn Boot Camp.
  • "Here at the Muscular Dystrophy Association, we stand hand in hand with Burn Boot Camp in our shared commitment to empower communities nationwide.
  • MDA and Burn Boot Camp will be posting throughout the campaign on social media using @MDAorg and @burnbootcamp with #BeTheirMuscle.

Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, March 21, 2024
HDAC, Gene expression, Cell, Disease, MDA, Associate, Patient, Research, DNA, Public policy, University, Muscular dystrophy, Doctor of Philosophy, Muscular Dystrophy Association, Duchenne muscular dystrophy, Clinical trial, Therapy, Histone deacetylase inhibitor, Stair climbing, Plasma protein binding, FDA, Steroid, DMD, Parent, Lancet, Pediatrics, Human, Food, Pharmaceutical industry

"The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.

Key Points: 
  • "The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.
  • “Muscular Dystrophy Association’s funding was fundamental for the discovery of HDACi in the treatment of Duchenne muscular dystrophy.
  • “We’re excited to celebrate this additional treatment option for people living with Duchenne muscular dystrophy.
  • My son is an example,” said Jessica Curran, MDA family member and mother of Conner who lives with Duchenne muscular dystrophy.

NFL Running Back Nyheim Hines Supports Nation's Largest St. Patrick’s Day Fundraising Campaign to Benefit Muscular Dystrophy Association

Retrieved on: 
Wednesday, March 13, 2024
Cleveland Browns, Nyheim Hines, ALS, MDA, Partnership, Muscular dystrophy, Research, Muscular Dystrophy Association, Family, NFL, Neuromuscular disease

Hines proudly wears the 2024 limited edition MDA Shamrocks t-shirt designed by MDA Board member Matthew Plummer who lives with neuromuscular disease.

Key Points: 
  • Hines proudly wears the 2024 limited edition MDA Shamrocks t-shirt designed by MDA Board member Matthew Plummer who lives with neuromuscular disease.
  • With family members who live or have lived with muscular dystrophy, Hines continues to generate awareness and raise critical funds for the neuromuscular disease community.
  • "It's truly humbling to continue to raise awareness for the neuromuscular disease community through the mission of Muscular Dystrophy Association.
  • It’s our privilege to collaborate with Nyheim to bring the fruits of our mission to his family and millions more across the country."

Scholar Rock Reports Full Year 2023 Financial Results and Highlights Business Progress

Retrieved on: 
Tuesday, March 19, 2024
Research, FDA, Clinical Trials, Cardiology, Biotechnology, Pharmaceutical, Health, Science, Oncology, Other Science, Overweight, SMN, Conference, Obesity, Keystone, MDA, Gilead Sciences, SAPPHIRE, SMA, Manuscript, Myostatin, Rock, Muscular dystrophy, Muscular Dystrophy Association, Trial of the century, Efficacy, Clinical trial, Therapy, Literature, Lunch, Pharmaceutical industry

“In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead.

Key Points: 
  • “In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead.
  • Scholar Rock remains on track to report topline data from the Phase 3 trial in the fourth quarter of 2024.
  • Scholar Rock also presented a literature review that describes the ongoing burden and significant unmet needs in SMA despite advancements in treatment.
  • Dr. Qatanani joined Scholar Rock in 2021 and has been leading all research functions since 2022 and driving Scholar Rock’s early-stage pipeline, including SRK-439.

Wilson Sporting Goods Joins Annual MDA Shamrocks Campaign with Introduction of Limited-Edition Glove

Retrieved on: 
Wednesday, March 13, 2024
Disease, ALS, MDA, Research, DNA, Sale, Muscular dystrophy, Muscular Dystrophy Association, Wilson Sporting Goods, Trust, Glove

CHICAGO, March 13, 2024 /PRNewswire/ -- Ahead of St. Patrick's Day, Wilson Sporting Goods proudly joins the Muscular Dystrophy Association (MDA) with the launch of the limited-edition 2024 MDA Shamrocks A2000® 1786 Glove.

Key Points: 
  • CHICAGO, March 13, 2024 /PRNewswire/ -- Ahead of St. Patrick's Day, Wilson Sporting Goods proudly joins the Muscular Dystrophy Association (MDA) with the launch of the limited-edition 2024 MDA Shamrocks A2000® 1786 Glove.
  • Wilson Sporting Goods joins annual MDA Shamrocks campaign with introduction of limited-edition glove.
  • Wilson Sporting Goods further supported MDA with a Giving Tuesday campaign before the introduction of the new glove this year.
  • The limited-edition 2024 MDA Shamrocks A2000® 1786 Glove will be available for purchase at participating sporting goods stores and online starting March 13 for $299.99.

Dyne Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Tuesday, March 5, 2024
Therapy, Myotonia, Central nervous system, Research, DYN, Dystrophin, Safety, DMPK, DMD, Exercise, Standard of care, Conference, DM1, Eteplirsen, Muscular Dystrophy Association, Fatigue, Myotonic dystrophy, Patient, Clinical trial, PMO, Male, G&A, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., March 05, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

NMD Pharma announces poster and oral presentations on skeletal muscle targeted ClC-1 therapy at two leading neuromuscular disease conferences

Retrieved on: 
Friday, March 1, 2024
EVP, Exercise, Hubli railway division, Conference, Phase II, SMA, Safety, Muscular Dystrophy Association, Science, Fatigue, Muscle weakness, Patient, NMD, Poster, Myasthenia gravis, Phase, MDA, Trial of the century, Congress, Pharmaceutical industry

ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise.

Key Points: 
  • ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise.
  • ClC-1 inhibition could be a possible mechanism to improve muscle fiber activation to address muscle weakness and fatigue in neuromuscular diseases.
  • Details of NMD Pharma’s poster presentations are below:
    The 4th Scientific International Congress on SMA is taking place in Ghent, Belgium, from 14-16 March.
  • : NMD Pharma be presenting one poster and one oral presentation at the congress.

Muscular Dystrophy Association Applauds Landmark Proposal on Accessible Air Travel at The White House

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Wheelchair, Disability, Aircraft, Air travel, Doctor of Philosophy, Air Carrier Access Act, Injury, Dehumanization, Muscular dystrophy, Safety, Empowerment, Muscular Dystrophy Association, ACAA, Joe Biden, Fear, Policy, DOT, Public policy, MDA, Travel, Airline

Donald S. Wood, PhD, President and CEO of the Muscular Dystrophy Association said, “I am immensely proud of the tireless efforts of MDA’s staff and advocates.

Key Points: 
  • Donald S. Wood, PhD, President and CEO of the Muscular Dystrophy Association said, “I am immensely proud of the tireless efforts of MDA’s staff and advocates.
  • This monumental proposal on accessible air travel reaffirms our commitment to serving families and we stand in solidarity with the disability community, championing inclusivity and accessibility in every facet of life.
  • This proposal represents a transformative stride towards empowering individuals to travel without the burden of fear, injury, or dehumanization."
  • Stay informed and engaged as we navigate the path towards a more accessible and inclusive world.

Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024
B cell, Therapy, Pharmacology, Diaphragm, Chemistry, Dystrophin, DMD, Part, Facial muscles, Duchenne muscular dystrophy, Exon, Conference, Wave, Health, Muscular Dystrophy Association, RNA, PN, Optimism, Protein, Oligonucleotide, RAPS, Half-life, MDA, Episcopal conference, Dietary supplement

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced its upcoming presentations at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL.

Key Points: 
  • Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study.
  • The presentations will also illustrate the impact of Wave’s novel PN chemistry on pharmacology of its exon skipping oligonucleotides.
  • These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle.
  • “At Wave, we increasingly continue to regard exon skipping as the preferred mechanism for altering DMD disease progression in those amenable to this approach.

Edgewise Therapeutics to Present on EDG-5506 for the Treatment of Becker Muscular Dystrophy at the 2024 MDA Clinical and Scientific Conference

Retrieved on: 
Wednesday, February 28, 2024
Biotechnology, Health, Pharmaceutical, Physical Therapy, Cardiology, BMD, Exercise, Principal investigator, Network, Myopathy, Edgewise, Conference, Muscular Dystrophy Association, University, Therapy, ARCH, Associate professor, University of Florida, Poster, Trade, Convention, MDA, Physiology, Episcopal conference, University of Copenhagen

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
  • The conference will take place at the Hilton Orlando in Orlando, FL from March 3-6, 2024.
  • Posters will be showcased during the Networking & Poster Reception in the Exhibit Hall on Monday, March 4, 2024, from 6 – 8 PM ET.
  • The Edgewise presentation and posters will be available on the Edgewise website when they are presented.