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Muscular Dystrophy News Today is providing virtual coverage of the Parent Project Muscular Dystrophy 2024 Annual Conference taking place June 27-30 in Orlando, Florida. Stay tuned to this page for all the latest news.

July 5, 2024 News by Marisa Wexler, MS

PPMD 2024: Gene therapy trials recruiting patients around the world

With the first gene therapy for Duchenne muscular dystrophy (DMD) now approved in the U.S. for most people with the disease, clinical trials are underway to better understand the safety and efficacy of…

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July 4, 2024 News by Marisa Wexler, MS

PPMD 2024: 4 therapies aiming to slow DMD progression now in trials

Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the progression of Duchenne muscular dystrophy (DMD), were spotlighted in a session at last…

July 3, 2024 News by Marisa Wexler, MS

PPMD 2024: Trials due for DMD gene therapies, cell regeneration

In its 30-year history, Parent Project Muscular Dystrophy (PPMD) has helped fund the development of many experimental treatments for Duchenne muscular dystrophy (DMD) and some of these are about to be tested…

July 2, 2024 News by Marisa Wexler, MS

PPMD 2024: DMD therapies to boost dystrophin advance in trials

More than half a dozen exon-skipping therapies for Duchenne muscular dystrophy (DMD) — all seeking to increase production of the dystrophin protein by modulating how the DMD gene is read — are now…

July 2, 2024 News by Marisa Wexler, MS

PPMD 2024: PTC to seek FDA approval of ataluren for DMD

Note: This story was updated Oct. 22, 2024, to remove a reference to the therapy being pulled from the market in Europe. Although this was the initial recommendation, the therapy has remained available…

July 1, 2024 News by Marisa Wexler, MS

PPMD 2024: Building community is vital for mental, physical health

Connecting with a supportive community is essential for people affected by muscular dystrophy to maintain both their emotional and physical well-being, according to Brandon Kozar, a psychologist at Nationwide Children’s Hospital. “When we come…

June 28, 2024 News by Marisa Wexler, MS

PPMD 2024: Conference keynote address recalls 30 years of advocacy

Parent Project Muscular Dystrophy (PPMD) held its first conference in 1994, in Orlando, Florida. A year later, at its meeting in Pittsburgh, fewer than two dozen people were in attendance, just a small…

June 27, 2024 News by Marisa Wexler, MS

PPMD 2024: 30 years of ‘progress, community, and resilience’

The Parent Project Muscular Dystrophy (PPMD) 30th annual conference kicks off today, offering families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) the chance to “celebrate three decades…

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