February 2024

PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization’s venture philanthropy program which provides industry funding to accelerate therapeutic development. This funding commitment aims to address current limitations in gene therapy delivery and will advance the development of the company’s…

Know More

With projects spanning gene therapy research, patient advocacy, and air travel safety, seven U.S. organizations will receive $140,000 in overall funding from the Muscular Dystrophy Associations (MDA) Advocacy Collaboration Grant Program. The program, which opened in 2022, seeks to support and enhance key public policy and advocacy initiatives through grassroot efforts. The overarching goal is to…

Know More

Pulling into a parking spot at my oldest son’s high school, I hit a parked car with my van. Thankfully, there was no damage to either vehicle, and the car’s owner, sitting in the front seat when I hit it, was amiable and understanding. I could safely unload my son Max, but I didn’t make…

Know More

“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…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More