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Changing society and its views on disability, one column at a time
Like me, my colleagues here at Bionews, the parent company of this website, write about their lives with chronic illness. In our columns, “we make a promise to our rare disease communities to deliver trustworthy information to help educate, engage, and champion the patient voice.” These words appear on the Bionews website, and they’re the…
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EMA agency continues to oppose renewing Translarna’s approval
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its recommendation not to renew conditional marketing authorization for Translarna (ataluren), a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The European Commission, which has final say over medication approvals in the European Union,…
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I wish all of my sons could have played football
I sat in the front row of the football stadium, watching my second-oldest son, Chance, 17, play football. It’s his senior year, and I’ve been front and center at each game. That front row has the accessible seating, which helps because Chance has three brothers living with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15,…
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CIFFREO Data Discussion Video
Watch the video HERE Pfizer recently provided this video updating the results from their Phase 3 micro-dystrophin gene therapy trial, CIFFREO, which were presented on October 12, 2024 at the World Muscle Society Conference in Prague. In this video, Francesco Muntoni, Chair of the CIFFREO trial steering committee, and Dan Levy, who leads DMD Clinical…
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An emergency preparedness plan is crucial for those with disabilities
As Western Pennsylvania natives, my wife, Wendy, and I are both Steelers fans. Naturally, when the Steelers played the Dallas Cowboys a couple weeks ago, we tuned in. If you’re not a football fan, you’re most likely unaware that the game was delayed for about an hour and a half as a huge thunderstorm rolled…
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RNA-based approach shows promise for DM1 in cell models
Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that this strategy potentially “could be beneficial…
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Gene therapy at low dose helps two OPMD patients with swallowing
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, according to an update from the therapy’s developer, Benitec Biopharma. Trial findings to date, which include reasonable treatment safety being seen, were presented at the…
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Catch Up on World Muscle Society 2024
Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. It offers a dynamic platform for sharing new data, fostering collaborations, and advancing the science behind therapies for diseases like Duchenne muscular dystrophy. This year’s event in Prague, Czech Republic, featured talks…
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WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)
Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined PPMD for a webinar during which Maitea Guridi, a Principal Clinical Scientist at Roche, shared learnings around the natural history of bone health in Duchenne. We discussed why fractures occur, with Maitea explaining that communication between muscles and bone is…
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Capricor now seeking FDA approval of DMD therapy deramiocel
Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy candidate to treat cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD), the company announced. Rolling submission means that Capricor will be submitting parts of the process to the U.S. Food and Drug Administration (FDA) as…
