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Cover of Alexander’s Not-So-Secret Diary, showing two cartoon boys, one seated in a wheelchair, with text introducing Alexander as a child born with a rare condition.

Patient Voices

New Digital Children’s Book, Alexander’s Not-So-Secret Diary, Now Available

Our new digital children’s book, Alexander’s Not-So-Secret Diary, brings this belief to life through the experiences of nine-year-old Alexander, who is living with Alexander disease (AxD), an ultra-rare, progressive and often fatal neurological disorder that affects approximately 1 in 1 to 3 million people worldwide.

Innovation at Work

Pioneering RNA-Targeted Medicines: What They Are and Why They Matter

RNA-targeted medicines work differently. Learn how they interact with RNA to address disease earlier than traditional approaches – and why it matters.

Illustration of RNA and RNA-targeted medicines alongside protein shapes, with one gray protein among yellow proteins

Innovation at Work

Hidden in Plain Sight: Four Takeaways on Addressing the Unseen Burden of Very High Triglycerides

More than three million people in the United States are living with very high triglycerides 500 mg/dL or higher, also known as severe hypertriglyceridemia (sHTG), yet many are left to navigate a complex care journey largely on their own. With support from Ionis, the Hidden in Plain Sight white paper was developed in partnership with the National Triglyceride Alliance, leading clinicians, patient advocates, people living with very high triglycerides and their care partners to examine the gap between recognition and care and the collective action required to close it.

“Hidden in Plain Sight: The Unseen Burden of Very High Triglycerides” with portraits of clinical contributors Ann Liebeskind, MD and Nishant Shah, MD.

Patient Voices

Finding Joy in the Journey: David and Melissa’s Life with Alexander disease (AxD)

For David and his mother, Melissa, moments of joy are an important part of life with Alexander disease (AxD), an ultra-rare, progressive and often fatal neurological disorder that affects approximately 1 in 1 to 3 million people worldwide.