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March 25, 2019March 25, 2019

Boys with Duchenne Muscular Dystrophy at High Risk for Bone Fractures

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Young boys with Duchenne muscular dystrophy (DMD) have a higher rate of bone fractures than healthy children, a retrospective study shows. The fractures are more frequent in lower limbs ... Read more

March 22, 2019March 22, 2019

CRISPR-Cas9 Corrects Common DMD-causing Mutation in Mice and Human Cells

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Restoring a functional dystrophin gene using the genome editing tool CRISPR-Cas9 corrected a common mutation behind Duchenne muscular dystrophy (DMD), exon 44 deletion, in mice and human cells, a study shows. The same research lab ... Read more

March 20, 2019March 20, 2019

New Enhanced Microdystrophin Versions May Help Restore Muscle Function in DMD, Mouse Study Suggests

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Engineered small versions of dystrophin protein, known as microdystrophins, have shown the ability to enhance muscle strength and function in preclinical models of Duchenne muscular dystrophy (DMD). Researchers from ... Read more

March 18, 2019March 18, 2019

Firefighters to Hike 130 Miles to Raise Awareness of Neuromuscular Diseases and Promote Newborn Screening

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Building on their international trade association’s relationship with the Muscular Dystrophy Association (MDA), two Virginia firefighters will set out on a 130-mile hike this month to raise awareness of ... Read more

March 15, 2019March 15, 2019

Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

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Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone — on heart and muscle health, but with less detrimental side effects, ... Read more

March 14, 2019March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of ... Read more

March 13, 2019March 13, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 13, 2019March 13, 2019

MDA’s 37th #MDAShamrocks Fundraiser is Underway

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To raise awareness and money to back efforts that help those living with muscular dystrophy (MD), the Muscular Dystrophy Association (MDA) has launched its venerable #MDAShamrocks campaign. More than ... Read more

March 11, 2019March 22, 2019

CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference

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CureDuchenne, one of the nation’s leading Duchenne muscular dystrophy (DMD) charities, is sponsoring 30 events across the U.S. this year to educate patients and their families about all aspects of ... Read more

March 8, 2019March 22, 2019

MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows

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All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational gene therapy MYO-101 in a clinical trial showed marked production of beta-sarcoglycan, the missing protein ... Read more

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