CureDuchenne Launches Supplemental Newborn Screening Initiative
CureDuchenne has launched a supplemental newborn screening initiative for Duchenne muscular dystrophy, in partnership with Brigham and Women's Hospital.
Category: Muscular Dystrophy
Muscular Dystrophy
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Muscular Dystrophy
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Impact of Chronic Inflammation May Spur New Duchenne Therapies
A new study reveals how chronic inflammation promotes muscle fibrosis, which could inform the development of new therapies for Duchenne muscular dystrophy (DMD), a fatal muscle disease.
Muscular Dystrophy
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FDA Approves Targeted Treatment for Duchenne Mutation
The US Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
Webinar: Vaccines and the Duchenne Community, Jan 22
As part of a new initiative centered on the COVID-19 vaccine rollout and the needs of the Duchenne community, CureDuchenne is hosting the following webinar on January 22: “COVID-19 Vaccines – What the Duchenne Community Needs to Know.”
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7th Annual STRIVE Awards for Duchenne Now Accepting Applications
PTC Therapeutics Inc is now accepting applications for the 2021 STRIVE Awards Program for Duchenne muscular dystrophy. The deadline to apply is March 15. Winners will be announced in September 2021.
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Pfizer Doses the First Participant in Duchenne Gene Therapy Study
Pfizer Inc announces that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of investigational gene therapy candidate PF-06939926 in boys with Duchenne muscular dystrophy (DMD).
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Parent Project Muscular Dystrophy Announces New Grant Opportunity
Members of Parent Project Muscular Dystrophy (PPMD)’s Certified Duchenne Care Center network have the opportunity to apply for a grant to support an inter-institutional collaborative project to focus on advancing the quality of care provided to Duchenne patients throughout the lifespan, PPMD announces.
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Dewpoint and Pfizer to Develop Potential Therapies for a Rare Form of Muscular Dystrophy
Dewpoint Therapeutics announces a new research collaboration with Pfizer for the development of potential therapeutics for the treatment of myotonic dystrophy type 1, DM1, a rare genetic disorder and one of two types of myotonic dystrophy.
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Divergent Genes Factor Into Duchenne Differences
Findings from a gene activity study published online recently in PNAS could lead to new treatments for Duchenne muscular dystrophy (DMD) and offer insights into factors that affect muscle development.
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Now Available: Duchenne Medical ID Bracelets
CureDuchenne announces a new initiative to deliver free medical ID bracelets to individuals in the United States living with Duchenne muscular dystrophy.
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PTC Therapeutics Hosts Translarna Deep Dive Webinar Dec 15
PTC Therapeutics Inc will host a webinar in its deep dive series discussing Translarna on Tuesday, December 15 at 12:00 noon ET.
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Powered Orthotic with Shoulder Assist Leads the Pack of New Products from Abilitech Medical
Abilitech Medical launches the Abilitech Assist, an assistive technology aid designed to help people with minimal strength resulting from neuromuscular disorders facilitate independent control of their arms by supporting and assisting both their shoulder and elbow.
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Grant Funds Next Step in Study to Assess Duchenne Progression Via Biomarkers
Duchenne UK, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) announce a joint grant of $686,500 to Dr William Evans (principal investigators) at the University of California, Berkeley to use a novel method to measure changes in total muscle mass in Duchenne muscular dystrophy patients through biomarkers in urine samples.
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PPMD Presents 1-Year Newborn Duchenne Screening Project Update
Parent Project Muscular Dystrophy (PPMD)’s Newborn Screening Pilot has been successfully screening babies born in New York State for Duchenne for over a year. Data were presented at the Association of Public Health Laboratories Newborn Screening Virtual Symposium about the ongoing pilot in New York State.
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Muscular Dystrophy Association Elects New President and CEO
The Muscular Dystrophy Association (MDA) has elected Donald S. Wood, PhD, as President and Chief Executive Officer, it announces. Dr Wood previously was Vice Chairman of the Board for the MDA and Vice President of Institutional Effectiveness at Odessa College.
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People Living with FSHD Tell Their Stories in New Report
The FSHD Society releases Voice of the Patient Report, a publication based on its June 29, 2020, externally led patient-focused drug development (EL-PFDD) meeting.
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Funding Aims to Advance Development of Duchenne Bone Wastage Therapy
CureDuchenne Ventures announces an investment in Mesentech Inc, a regenerative medicine company with a prodrug platform that selectively delivers therapeutics to bone. The investment is part of a new joint funding collaboration with the Charles H. Hood Foundation (CHF) that looks to advance early-stage research for pediatric conditions.
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Cystic Fibrosis Gene’s Duchenne Effect
A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), UT Southwestern researchers suggest, in the Journal of the American Heart Association.
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