News

March 29, 2019March 29, 2019

Santhera Filing for EMA Conditional Marketing Authorization for Idebenone to Treat Respiratory Issues in DMD

News

Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory ... Read more

March 27, 2019March 27, 2019

PPMD, Cure SMA Join Prometheus in Data Collection Partnership

News

Cure SMA and Parent Project Muscular Dystrophy (PPMD) have formed a strategic partnership with Prometheus Research to improve data collection and analysis for patients with spinal muscular atrophy (SMA) and Duchenne muscular dystrophy ... Read more

March 25, 2019April 18, 2019

Boys with Duchenne Muscular Dystrophy at High Risk for Bone Fractures

News

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, 2019April 18, 2019

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

News

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, 2019April 5, 2019

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

News

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 29, 2019

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

News

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 29, 2019

Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

News

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 27, 2019

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

News

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 28, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News

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 28, 2019

MDA’s 37th #MDAShamrocks Fundraiser is Underway

News

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

Pin It on Pinterest