Sloane was born with a KIF1A mutation and is a special case at n-Lorem—she was the first patient to receive a medicine originally designed for someone else. Her mother, Megan, and grandfather, Tom, join the Patient Empowerment Program to share her journey and provide an update on how she’s doing just over six months after treatment.

This episode is proudly sponsored by Hongene Biotech

• Sloane’s KIF1A diagnosis
• Nano-rare diseases affect entire families
• Sloane was able to use the same ASO as another KIF1A patient, Susannah
• Finding n-Lorem through KIF1A.ORG
• Shocked that n-Lorem was able to create personalized treatments efficiently at cost
• Sloane’s case is unique in a variety of ways and the decision to treat
• How Sloane has responded to treatment
• Are better measurements of benefit needed for nano-rare patients?
• How Sloane has brought empathy, joy, and positivity to her family
• Tom unretired to help fund research and support
• The origins of ASO medicine is a long haul

Megan Hedstrom is mom to three with husband Kyle: Griffin, Declan, and Sloane. Sloane was diagnosed with KIF1A Associated Neurological Disorder in August 2021 and she started receiving her ASO treatment from N-Lorem in May of 2024. Megan and her family live in Scottsdale, Arizona where she works in investment real estate.

Tom Lowe is a KIF1A.ORG supporter and grandfather to Sloane, another KAND superstar. Tom has spent his career in business running the real estate and private equity operations for his family’s office in Arizona.

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We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.