- A week in the life of an overwhelmed caregiverby Betty Vertin on February 14, 2025 at 3:00 pm
I’ll be straightforward about caregiving: It’s exhausting. I’m the mother of seven children: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3. I’m also The post A week in the life of an overwhelmed caregiver appeared first on Muscular Dystrophy News.
- Phase 1 trial of SAT-3247 finishes enrolling healthy volunteersby Marisa Wexler, MS on February 13, 2025 at 1:00 pm
A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of The post Phase 1 trial of SAT-3247 finishes enrolling healthy volunteers appeared first on Muscular Dystrophy News.
- It started with shoes, but now everything is too heavyby Robin Stemple on February 12, 2025 at 3:00 pm
One thing I was born with, aside from my facioscapulohumeral muscular dystrophy (FSHD), was narrow feet. As part of a working-class family, where money always seemed to be in short The post It started with shoes, but now everything is too heavy appeared first on Muscular Dystrophy News.
- World of Warcraft event raises $2M to support DMD research, careby Marisa Wexler, MS on February 11, 2025 at 1:15 pm
A fundraiser through the online role-playing game World of Warcraft has raised more than $2 million to support CureDuchenne, an organization dedicated to combating Duchenne muscular dystrophy (DMD). The fundraiser, The post World of Warcraft event raises $2M to support DMD research, care appeared first on Muscular Dystrophy News.
- Growing into limb-girdle muscular dystrophy — and, better, marriageby Patrick Moeschen on February 10, 2025 at 3:00 pm
I’ve been married for 11 years. Before then, as I grew up with what I eventually discovered was limb-girdle muscular dystrophy, I learned a lot about life, perseverance, and relationships. During The post Growing into limb-girdle muscular dystrophy — and, better, marriage appeared first on Muscular Dystrophy News.
- Phase 2 trial shows DMD treatment ifetroban boosts heart functionby Marisa Wexler, MS on February 6, 2025 at 1:00 pm
Treatment with oral therapy ifetroban led to improvements in measures of heart function in people with Duchenne muscular dystrophy (DMD), according to top-line results from a clinical trial. The “impressive The post Phase 2 trial shows DMD treatment ifetroban boosts heart function appeared first on Muscular Dystrophy News.
- Exploring new possibilities in the realm of art and disabilityby Shalom Lim on February 5, 2025 at 3:00 pm
The Lunar New Year has always been a time of reflection and renewal for me. This year, though, it was bittersweet because my longtime caregiver plans to return to her The post Exploring new possibilities in the realm of art and disability appeared first on Muscular Dystrophy News.
- Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathyby Parent Project Muscular Dystrophy on February 4, 2025 at 5:56 pm
Cumberland Pharmaceuticals Inc. has announced positive top-line results from its Phase 2 FIGHT DMD trial evaluating ifetroban for the treatment of Duchenne cardiomyopathy. Ifetroban is an oral thromboxane receptor antagonist aimed at reducing inflammation and… The post Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathy appeared first on Parent Project Muscular Dystrophy.
- Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UKby Margarida Maia, PhD on February 4, 2025 at 2:15 pm
Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of ENTR-601-44, at increasing doses, in Duchenne muscular dystrophy (DMD) patients with a The post Phase 1/2 trial of exon 44 skipping therapy for DMD cleared in UK appeared first on Muscular Dystrophy News.
- To a parent, the years always seem too shortby Betty Vertin on January 31, 2025 at 3:00 pm
I read a quote the other day on Facebook: “When you are raising babies, you feel how long the days are. When raising teens, you feel with every single bone The post To a parent, the years always seem too short appeared first on Muscular Dystrophy News.
- MDA honors researcher, patient advocate with 2025 Legacy Awardsby Marisa Wexler, MS on January 30, 2025 at 8:15 pm
The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The post MDA honors researcher, patient advocate with 2025 Legacy Awards appeared first on Muscular Dystrophy News.
- Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYSby Parent Project Muscular Dystrophy on January 27, 2025 at 7:14 pm
Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four… The post Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS appeared first on Parent Project Muscular Dystrophy.
- Update from PPMD’s 2025 Duchenne Healthcare Professionals Summitby Parent Project Muscular Dystrophy on January 23, 2025 at 5:22 pm
PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the FDA, NIH, CDC, and international representatives in one room to discuss the latest updates in dystrophinopathy care and research at… The post Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit appeared first on Parent Project Muscular Dystrophy.
- Race to End Duchenne Kicks Off 20th Anniversary at Walt Disney World Marathon Weekendby Parent Project Muscular Dystrophy on January 17, 2025 at 7:07 pm
The Race to End Duchenne program is celebrating our 20th anniversary in 2025, and we spent the weekend of January 8-12 kicking it off at the place where it all began – the Walt Disney… The post Race to End Duchenne Kicks Off 20th Anniversary at Walt Disney World Marathon Weekend appeared first on Parent Project Muscular Dystrophy.
- PPMD Advocacy in the 119th Congress and the 47th Administrationby Parent Project Muscular Dystrophy on January 16, 2025 at 2:37 pm
As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed to furthering the needs of our community through comprehensive, creative, and tenacious advocacy on Capitol Hill, regardless of politics. Over… The post PPMD Advocacy in the 119th Congress and the 47th Administration appeared first on Parent Project Muscular Dystrophy.
- Advance the Research Evolution with The Duchenne Registryby Parent Project Muscular Dystrophy on January 16, 2025 at 1:59 pm
Everyone can help advance the research evolution for Duchenne and Becker. Bringing data sources together from multiple research endeavors is a key component of PPMD’s plan to advance the research evolution, and The Duchenne Registry… The post Advance the Research Evolution with The Duchenne Registry appeared first on Parent Project Muscular Dystrophy.
- PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, Californiaby Parent Project Muscular Dystrophy on January 15, 2025 at 2:23 pm
PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, California, January 15-17. Since the Summit’s inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders… The post PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California appeared first on Parent Project Muscular Dystrophy.
- Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolutionby Parent Project Muscular Dystrophy on January 10, 2025 at 3:50 pm
In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that helps track what happens when individuals are on more than one therapy and how we can continue to evolve standards… The post Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolution appeared first on Parent Project Muscular Dystrophy.
- Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044)by Parent Project Muscular Dystrophy on January 9, 2025 at 5:00 pm
Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen (del-zota) for treatment of individuals living with Duchenne muscular dystrophy… The post Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044) appeared first on Parent Project Muscular Dystrophy.
- PPMD’s Race to End Duchenne Program Celebrates 20 Years of Going the Distance to End Duchenneby Parent Project Muscular Dystrophy on January 8, 2025 at 2:38 pm
PPMD is celebrating the 20th anniversary of our endurance fundraising program, Race to End Duchenne, this weekend in Orlando, Florida. 185 team members will participate in the 2025 Walt Disney World® Marathon Weekend, presented by… The post PPMD’s Race to End Duchenne Program Celebrates 20 Years of Going the Distance to End Duchenne appeared first on Parent Project Muscular Dystrophy.
- New study uncovers key insights into protein interactions in Duchenne muscular dystrophy, paving way for more targeted therapieson December 31, 2024 at 6:41 pm
A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD).
- New drug shows promise against Duchenne muscular dystrophyon December 16, 2024 at 5:56 pm
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound called K884 can boost the natural repair abilities of muscle stem cells. Current treatments can slow muscle damage, but don’t address the root problem.
- New discovery enables gene therapy for muscular dystrophies, other disorderson November 14, 2024 at 9:11 pm
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
- Mouse model reveals liver involvement in muscular dystrophyon October 24, 2024 at 6:55 pm
A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 — the most prevalent form of adult-onset muscular dystrophy — provides insight into why patients develop fatty liver disease and display hypersensitivity to medications, making treatment difficult. The new model opens avenues for screening new medications for liver toxicity prior to patient trials.
- New gene therapy approach shows promise for Duchenne muscular dystrophyon July 24, 2024 at 11:12 pm
Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).