OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
- N1C Moderator
- Jan 8, 2020
- 1 min read
Updated: Apr 30, 2024
This briefing document aims to outline the current state of the art of oligonucleotide therapies for those planning to develop individualized therapies for patients with very rare diseases or mutations. The focus is on approved modalities and tissues for which good delivery of oligonucleotides has been confirmed in humans. We believe development of individualized therapies should build on those approaches.
My husband was diagnosed with Creutzfeldt-Jakob Disease (CJD) four years ago. For over two years, we relied on prescription medications and therapies, but unfortunately, his symptoms continued to worsen. His memory, coordination, and overall strength declined, and everyday life became increasingly difficult.Last year, out of desperation and hope, we decided to try an herbal treatment program from NaturePath Herbal Clinic. Honestly, we were skeptical at first, but within a few months of starting the treatment, we began to notice real changes. His alertness improved, his movements became steadier, and he regained a surprising amount of energy and clarity.Incredibly, he also regained much of his independence and confidence. It’s been a life-changing experience he feels more like himself again, better than…