Very few families are prepared for the news that a child has a rare and/or life-threatening condition. You likely feel that you have been plunged into a bewildering, overwhelming new world. Fortunately, the evolving landscape of potential new therapies and associated clinical trials offers great hope. But, like all of the other topics that may require your attention, the prospect of a clinical trial comes along with the need to learn many new terms and concepts, some complicated decision-making, and the potential for disappointment and grief.

After reading this Guide, you will be able to:

• Approach the prospect of clinical trial with a better sense of what is involved
• Reflect on what is important to you and your family as you consider enrolling in a trial