Giving Tuesday 2024: Fuel Our Work

Today is #GivingTuesday - a day dedicated to making an extraordinary impact. For families battling rare neuromuscular diseases, hope often feels just out of reach. But together, we can change that.  

This year, thanks to supporters like you, we’ve made remarkable progress.  This includes developing advanced therapies to target rare genetic mutations and preparing several promising treatments for clinical trial.

Behind every achievement is a family waiting for a breakthrough. A child waiting for a chance. We can’t stop now.

This Giving Tuesday, your generosity will go TWICE as far.

Thanks to the generosity of Kindness Over Muscular Dystrophy, every dollar donated will be matched up to $100,000 through the end of the year.  

Your gift today will accelerate life-changing research and bring us closer to the therapies patients and their families have been waiting for.  

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Donate Today

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This isn’t just about funding research—it’s about giving hope. It’s about providing a lifeline to those who thought they were out of options.  

Together, we can make the impossible, possible.

Together, we are building a world where rare doesn’t mean impossible. Thank you for being the light that guides us toward that future.

To learn more about our scientific progress, keep reading below.

With your support, we will continue to make scientific breakthroughs and bring hope to those desperately waiting for treatment.

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Donate Today

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Progress in 2024

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• Further development of next generation AAV capsids, including the completion of a non-human primate study. This study identified a next-gen AAV candidate that avoids off-target distribution to the liver which maintaining equal to or greater distribution to the muscle. This development is a critical achievement to advance each of our gene therapy programs aimed at treating neuromuscular diseases.
• Completion of the preIND for one of our DMD programs targeting a rare mutation, which demonstrated alignment with the FDA regarding non-clinical, manufacturing and clinical design.
• Completion of exploratory pharmacology studies for 2 rare DMD mutations.
• Completion of the development of humanized DMD mouse models for 5 DMD mutations.
• Completion of cell line corrections of 3 additional rare DMD mutations.
• Completion of the preIND for our SCA3 program and began preparation for large-scale manufacturing of the therapeutic.
• Completion of the preIND for our LGMD2i program and completed a long-term efficacy and pharmacology study. These studies showed long-term expression of the FKRP transgene and sustained functional rescue in the FKRP mouse model.
• Completion of a year-long efficacy and pharmacology study, and a 12-week study for our LGMD2g program, which resulted in robust TCAP expression in diseased TCAP mouse model.
• Completion of pharmacokinetic in-vivo study for our LGMD2b program, which validated our previous protein optimization studies designed to extend half-life, expression and efficacy.

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With your support, we will continue to make scientific breakthroughs and bring hope to those desperately waiting for treatment.

Donate Today