Five Rare Disease Toolkits Every Patient and Parent Should Read

As A Rare Disease Patient or Parent Advocate, we know that so often you’ve felt an overwhelming frustration with the lack or resources out there and thought: hasn’t anyone been here before? Why can’t someone show me the way?

This thought is exactly why we undertook the long and well-researched project of gathering information from across the Rare Community to bring to life our series of RARE Toolkits.

While more are being created each day, here are the top 5 RARE Toolkits for you to start reading:

1. Parenting a Child With a Life-Limiting Illness : This is an extremely useful guide for patients and their families. After learning the typical prognosis for the disease, it is not unusual to worry about the future and anticipate what’s to come. But with a slowly regressing illness, there is a lot of time to understand what you will need medically and emotionally. There will be time to connect with others and gain support; there will be time to explore treatment methods.
2. Taking Control: Gaining Independence as a Young Adult with a Rare Disease: Becoming an adult is often marked by changes and challenges. Teens with rare or undiagnosed diseases know this all too well. And although they may know a lot about their conditions, many struggle with moving towards greater independence. This toolkit will support rare disease families and their children who are approaching adulthood by covering the steps they should take to become more independent, strong, and proactive individuals.
3. Genetic Testing: Is This My Path to a Diagnosis: This toolkit is designed to provide rare disease patients and their families an introduction to genetics and genetic testing. Advances in genetic testing are rapidly changing the way patients are being diagnosed and treated and providing new hope to patients with rare, genetic diseases.’
4. Access to Unapproved Medicine: Is This an Option For Me? (USA) For many patients, access to a medicine outside the clinical trial or commercial setting can represent a new and, in many cases, life-saving treatment option. The process of obtaining treatments that are not currently available can be confusing for many patients and their families. In this toolkit, we will introduce what access programs are, how they work, and what patients and their families can do to further explore.
5. Bringing Rare Disease To Capitol Hill: Advocating for Your Cause Capitol Hill may seem like an enigma to some. Shrouded in mystery, its activities may seem elusive and arcane. How does it function? How can it benefit you? Many are not aware what an incredible resource Capitol Hill can be for the rare disease community. Laws and bills go before Congress and the Senate every day that can directly impact the rare disease community—as a result of advocates like you! From diffusing just how a bill becomes a law to understanding the intricacies of Congress, this toolkit will help integrate you into the public’s role in government.