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Why patient advocacy is important for those of us with Duchenne
Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate Connection event (SPACe) hosted by the SingHealth Patient Advocacy Network (SPAN). As a cast member of the incredible SPANtastic Theatre plenary skit team, I not only had the chance to perform, but also to experience something deeply transformative. Although I’ve…
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Losmapimod fails to significantly improve FSHD outcomes: Study
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD), according to top-line data from the REACH Phase 3 trial. Participants given losmapimod did see improvements over 48 weeks, but the placebo group didn’t show a…
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Navigating Access & Resources
Navigating Access & Resources Navigating insurance access for medical care such as approved therapies or equipment can be daunting. Parents, patients, and healthcare providers spend hours putting together documents and speaking on the phone trying to convince payers to approve what is needed to keep us, our children, or our patients, as healthy as possible.…
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A friend in the DMD world makes a difference in my self-care
In my recent columns, I’ve shared several challenges we’ve faced in the past months of my family’s journey with Duchenne muscular dystrophy (DMD). My accessible van will be in the shop for the foreseeable future. I’ve returned to work after over a decade at home as a mom to several children and a caregiver to…
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Atamyo, Dion Foundation partner to expand LGMD study into US
Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5. The Phase 1b study (NCT05973630), designed to evaluate the safety, efficacy, and pharmacological and immunological properties of ATA-200,…
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NS Pharma DMD treatment gets FDA rare pediatric disease status
The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted this designation are aimed at serious or life-threatening diseases affecting children and fewer than 200,000 U.S. patients. “We are grateful for this designation, which can help us accelerate the…
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Watch the Recording: Advocating for A Successful School Year
Recently, PPMD’s Alexis Hazlett was joined by panelists Jodi Krause (Colorado Children’s Hospital), Jill Castle, and Patrick Moeschen to discuss advocating for your child throughout the school year. The speakers dove into the differences between IEP (Individualized Educational Plan) and 504 plans, understanding your child’s educational rights, and shared tips to enhance communication and working…
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How my late brother and I left a mark on our DMD community
While observing World Duchenne Awareness Day over the weekend, I reflected on the journey my family and I have taken, which has been shaped by Duchenne muscular dystrophy (DMD). This year the Muscular Dystrophy Association (Singapore), or MDAS, celebrated the day during its annual Go the Dystance carnival. Growing up with DMD, I found that…
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Teaching students about life with limb-girdle muscular dystrophy
As most of America’s children go back to school, I thought it’d be timely to offer readers my perspective, as a retired teacher and wheelchair user, on the beginning of an academic year. I began teaching middle school music and band in the fall of 1995. I was 22 years old and had just graduated…
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DYNE-251 leads to improvements in motor function in DMD: Update
Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne is launching registrational groups of trial participants, the…
