Advancing research in gene therapy: Supporting patients, caregivers, and advocacy communities
Bringing gene therapeutics to patients with diseases that have a high unmet medical need fuels our research and development pipeline. We have active preclinical and clinical programs for neuromuscular, metabolic, cardiovascular and central nervous system conditions, with particular attention on rare and more common diseases.
About AskFirst™
AskFirst™ is driven by our mission to improve patients’ quality of life and our desire to bring innovative genetic medicines to market.
Our outreach initiative is designed for advocacy groups, patients, and caregivers seeking information about ongoing developments in gene therapy research. We believe collaboration and education are essential to advancing scientific understanding, and that patients and their support systems play an important role throughout the research process. Together, we engage with stakeholders to support progress in science, technology, regulatory understanding, and public policy related to genetic medicine. From informing clinical study design to engaging with research teams, our commitment to patient advocacy remains central to everything we do.
Patient voices are instrumental in driving the innovation and progress that define AskFirst™. We’re applying collaboration and compassion to eradicate diseases.
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“We put a lot of eggs in this basket of hope. To those working on a treatment for LGMD, time is of the essence. We are desperate for anything that might improve our sons’ daily lives.”
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Ask Kara
“In the dynamic field of gene therapy for complex diseases, the voices and
experiences of individuals and their families drive our mission to innovate,
collaborate, and ultimately, transform lives.”
Kara Witcoff
Patient Advocacy & External Engagement
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