Acknowledgements

This book exists because families take part in research. My first thanks go to the participants, caregivers, and people living with rare and neurodegenerative conditions who have answered the questions, uploaded the reports, and told their stories, often in the hours their own lives allowed and often for the benefit of someone else's child.

Thank you to the study coordinators I have worked with and learned from, who showed me that the relationships are the study, and to the patient advocacy group leaders who brought me into their registries and their communities and told me plainly what was not working.

Thank you to my colleagues and collaborators in ALS research, in the rare epilepsy community, and in the OHDSI and open science communities. Their weekly meetings, careful questions, and shared code taught me much of what is in these pages.

Thank you to the readers who contribute case studies, rules, and corrections to this living book. Contributors who wish to be named are listed here as each version is published.

Finally, thank you to Jim and to our children, and above all to Charlie, who is the reason I learned any of this.