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Insights by Ira: Mission Complete
Just four days prior to my inaugural public engagement as National Ambassador in 2024, I began not feeling well. Confronted with this situation, I was faced with two options: notify my MDA contact regarding my potential absence from the highly anticipated event or adhere to my established practice of persevering through challenges in order to…
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Dyne, citing positive trial data, to ask FDA to approve DMD therapy
Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, after a Phase 1/2 trial showed encouraging results. Data from the registrational expansion cohort of the DELIVER trial indicated that DYNE-251 is capable…
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My hope for the FSHD community this Advent season
Advent is the beginning of the church year for Christians. What better way to start the year than with a season of hope? During Advent, Christians prepare for the coming of Jesus, whom we believe is the incarnation of God. He entered our world, becoming human, when he was born in Bethlehem. It’s no accident…
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Dyne announces positive topline results from Phase 1/2 trial of z-rostudirsen in Duchenne amenable to skipping exon 51
As an early investor in Dyne Therapeutics, we are pleased to share their exciting news that skipping exon 51 in the dystrophin gene with z-rostudirsen in the registrational expansion cohort met the primary endpoint–a statistically significant increase in dystrophin at 6 months—and was also associated with functional improvements in multiple measurements. The DetailsThe DELIVER trial…
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New trial results highlight gains in arm and heart function for DMD
In the Phase 3 HOPE-3 clinical trial, the experimental cell therapy deramiocel outperformed a placebo at improving arm function and heart health in people with Duchenne muscular dystrophy (DMD), according to top-line results announced by its developer, Capricor Therapeutics. Capricor has said it plans to use the HOPE-3 data as a basis to resubmit its…
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Satellos receives clearance by FDA to initiate Phase 2 study in Duchenne
Satellos Biosciences has received Investigational New Drug (IND) clearance from the FDA, as well as other global regulatory agencies, to conduct a 3-month placebo-controlled Phase 2 study of SAT-3247 in ambulatory individuals with Duchenne. SAT-3247 is an oral, small molecule drug aiming to regenerate skeletal muscle that is lost in Duchenne. Satellos expects the first…
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In Case You Missed It…
Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
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Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne
Dyne Therapeutics has announced positive topline results from its Registrational Expansion Cohort (REC) of the Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in people living with Duchenne who are amenable to exon 51 skipping. Z-rostudirsen is an exon skipping product designed to enable targeted muscle tissue delivery and promote exon…
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Finding the right words to describe our life experiences
Words matter. For that reason, I’d like to share my thoughts on language, chronic illness, and how words shape societal views. I believe the way we use language can improve how patients living with rare conditions are seen, understood, and treated by the world at large. I’ve lived with limb-girdle muscular dystrophy all my life…
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Clinical Research Alert: Phase 1b Study of SRD-001 in Adults with DMD-Related Cardiomyopathy
Researchers at Sardocor Corp are seeking adults with cardiomyopathy (heart disease) secondary to Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial to evaluate the safety and explore the efficacy of the investigational gene therapy SRD-001 (AAV1/SERCA2a). In heart failure, the body does not make enough of the SERCA2a protein, which helps heart…
