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“So, are you dating anyone?” Could there possibly be five more scary words spoken to a single person? Finding love is hard for anyone and can be especially tricky if you have a disability, however, it is not impossible, says dating coach Erika Ettin. As Valentine’s Day approaches, we talked with Erika (@alittlenudge) and Ryan…

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PPMD recently hosted our fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, FL. Over the course of three impactful days, we were joined by around 300 healthcare professionals, researchers, and industry partners to collaborate, connect, and share knowledge, with the goal of learning from one another to ensure that caring for individuals with the latest…

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Dyne Therapeutics recently joined PPMD for a community webinar to present initial clinical data from the DELIVER trial of DYNE-251, a next-generation exon skipping investigational product, in individuals with Duchenne who are amenable to exon 51 skipping. The Dyne team highlighted that the safety profile for DYNE-251 is favorable, and pointed to the company’s proprietary…

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REGENXBIO Inc. has announced that enrollment has completed at dose level 2 of the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place…

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It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) lose upper body function and the ability to breathe on their own, according to a study by researchers of the HERCULES project, which set out to construct a natural history model of the disease to support cost evaluations of new…

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Amid a topsy-turvy start to 2024, I’ve marked a date on my calendar I’ve been looking forward to more than any other: Valentine’s Day. While it might not be a particularly significant day for many others, it’s a big deal for me. It’ll be my first time celebrating the day after unexpectedly falling in love…

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RegenXBio shared positive updates from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial for individuals with Duchenne aged 4-11 years old.  The 3 individuals in the lowest dose cohort all demonstrated reductions in CK levels, and expression of micro-dystrophin averaging 44% at 3 months.  Enrollment is also complete for a second cohort, which will…

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I do too much. You might assume I’m talking about caregiving because you’re reading a column written by a caregiver. And in part, you’re right. But mostly, it’s an admission that I’ve been doing things for my three sons with Duchenne muscular dystrophy (DMD) that they can do themselves. My oldest son, Max, 18, is…

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A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company appealed the negative opinion issued in September 2023…

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Earlier this week, Duchenne parent and PPMD Advocate Jill Castle testified before the Arizona State Senate Health & Human Services Committee in support of Senate Bill 1020, which seeks to add Duchenne to Arizona’s newborn screening panel. PPMD, along with partner organizations, also signed onto a letter in support of SB 1020. We’re excited to…

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