Eighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient Academy — a three-day series of master classes aimed at deepening global understanding of Duchenne muscular dystrophy (DMD) and coordinating advocacy strategy. The Dec. 6-8 conference, an annual event since its 2017 start, covered subjects…
Orsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) to treat people with Duchenne muscular dystrophy (DMD) responsive to exon 53 skipping…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
Expecting to award roughly $18 million in grants in 2020 to support research across neuromuscular diseases, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better treat and otherwise ease life for those touched by muscular dystrophy and numerous other, often rare, illnesses.
Gene therapies — after a tragedy kicked them back to the lab some two decades ago — are beginning to come into their own, making possible work in gene-targeted and combination treatments for neuromuscular diseases once unimaginable. But many challenges — from the likelihood of no “one and done” gene…
Blocking the protein receptor IGF2R could promote muscle regeneration and improve muscle strength in people with Duchenne muscular dystrophy (DMD), a study in mice suggests. The study, “Blockade of IGF2R improves muscle regeneration and ameliorates Duchenne muscular dystrophy,” was published in the journal EMBO…
The best relationships aren’t full of luxurious vacations and daily smiles and laughter. Long-lasting couples have been to hell and back together, growing closer and stronger through the storms. Ups and downs because of illness and vulnerabilities, along with the need for extra support, can test an interabled relationship. But…
The gene-editing tool CRISPR/Cas9 was used to correct defects in the DMD gene and restore dystrophin protein production, lengthening the lives of pigs in a model of Duchenne muscular dystrophy (DMD) and altering heart cells from a patient to make them less prone to irregular beats, researchers report. The scientists…
Life with Duchenne muscular dystrophy is far from predictable. We can’t foresee how our bodies will change. When I turned 18 (I’m 23 now), I didn’t know that five years later, I would need help showering and getting off the…
Longtime Muscular Dystrophy Association (MDA) supporter CN Brown Company, a CITGO Petroleum Corporation (CITGO) marketer, raised more than $300,000 for the MDA’s Maine chapter in 2019. “Our family’s commitment to helping win the fight for families affected by MDA has remained strong for generations,” Jeff Jones, CN…
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