CureDuchenne and Pfizer Discuss Opening Trial Sites in Phase 3 CIFFREO Study of Gene Therapy
CureDuchenne released a webinar on the upcoming opening of U.S. trial sites for Pfizer's CIFFREO trial, a phase 3 study of a gene therapy.
Category: Muscular Dystrophy
Muscular Dystrophy
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Muscular Dystrophy
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FSHD Society Deploys AI Technology to Improve Clinical Trial Design
The FSHD Society is launching a collaboration involving CTRN and BullFrog AI to analyze a dataset collected from patients with FSHD.
Muscular Dystrophy
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Clene Nanomedicine Presents Updated Clinical Data at MDA Conference
Clene Nanomedicine Inc presents results from Phase 2 RESCUE-ALS and REPAIR trials and preclinical data in ALS at the 2022 MDA Conference.
MDA and Filmmakers Launch Fundraiser to Send Kids to Camp
Justin Skeesuck and Patrick Gray’s award-winning film I’ll Push You is available for a virtual nationwide screening now through January 30 as part of a Muscular Dystrophy Association (MDA)’s fundraising effort to send disabled kids to summer camp.
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Apply Now for 2022 STRIVE Grants for Duchenne Projects
PTC Therapeutics Inc announces the launch of the eighth annual STRIVE awards program for Duchenne muscular dystrophy. The 2022 application process is now open to patient advocacy organizations.
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New Gene Therapy Approach Offers a Potential Long-Term Treatment for Limb-Girdle Muscular Dystrophy 2B
Children’s National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease.
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CureDuchenne Commits $1 Million to Gennao Bio to Accelerate Duchenne Research
CureDuchenne Ventures announces a $1 million investment in Gennao Bio. The investment aims to expand nucleic acid therapeutics toward targeting muscle diseases.
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Stem Cell Therapy for Duchenne Muscular Dystrophy Described in Recent Study
Promising animal studies and earlier investigations led to a trial of intravenous stem cell therapy in an 11-year-old boy with Duchenne muscular dystrophy, described in the winter issue of the Journal of American Physicians and Surgeons.
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MDA Awards 18 New Grants, Totaling More Than $1.6M
The Muscular Dystrophy Association (MDA) announces the awarding of 18 new MDA grants totaling over $1.6 million toward neuromuscular disease research.
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Finding Accessible Employment in the Work-from-Home Era
As part of National Disability Employment Awareness Month, Muscular Dystrophy Association announces a DEI Coalition that will facilitate people with disabilities entering the workforce.
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Parent Project Muscular Dystrophy Completes Newborn Screening Pilot Program
Parent Project Muscular Dystrophy announces that its Newborn Screening Pilot has successfully reached its completion, screening more than 36,000 babies born in New York State over the last 2 years.
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Mount Sinai Opens Charles Lazarus Children’s Abilities Center
Mount Sinai announces the opening of The Charles Lazarus Children’s Abilities Center, which aims to transform and shape the course of children’s rehabilitation.
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Register Now for 2022 MDA Clinical & Scientific Conference
For the first time, the annual conference will be presented both in-person in Nashville, TN, and virtually on March 13-16, 2022.
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WAE, Whizz-Kidz and Frazer-Nash Consultancy Join Forces on Innovative New Wheelchair
Whizz-Kidz has retained Williams Advanced Engineering (WAE) to refine the DREAM wheelchair concept, which has been designed by children for young wheelchair users.
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Scientists Offer New Hope for the Treatment of Fukuyama Congenital Muscular Dystrophy
Scientists rescue Fukuyama congenital muscular dystrophy-related brain defects in a new organoid model developed using stem cells from affected patients.
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CureDuchenne FUTURES Conference To Be Conducted Virtually October 8-10
CureDuchenne announces the annual FUTURES National Conference from October 8-10, 2021, will be hosted virtually this year.
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Sarepta Therapeutics Announces Scholarship Recipients
Sarepta Therapeutics Inc announces 15 recipients of Route 79, The Duchenne Scholarship Program created to recognize exceptional individuals living with Duchenne muscular dystrophy as they pursue their post-secondary education.
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PTC Therapeutics Recognizes STRIVE Awards Recipients for 2021
PTC Therapeutics Inc announces the three recipients who will receive funding through the company’s STRIVE Awards program. The global STRIVE Awards program provides grants to non-profit organizations serving the Duchenne muscular dystrophy community.
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