At the National Organization for Rare Disorders (NORD) Rare Disease & Breakthrough Summit in Washington D.C., Rare Disease Report spoke with Lesley Bennett who leads Connecticut’s Rare Action Network on behalf of NORD.
As Bennett explains in this interview, the Rare Action Network includes anyone interested in rare diseases (patients, their families, health care providers, companies, legislators) and their purpose is to raise awareness of rare diseases and to work with the legislature on issues that affect patients within their state.
Bennett said, “a lot of our patients have problems getting access to services because people don't understand the disorders. We don't fit into any box and it's been difficult making people aware of that. For the last 10 years we've been working to get an rare disease Advisory Council to the Connecticut legislature and the Connecticut legislature was so confused by this request that they developed a task force.” The task force has 4 more meetings before concluding in January and it is being used to expose the legislature to different issues facing the rare disease community.
On a personal note, Bennett’s daughter has a rare metabolic disorder that has left her wheelchair-bound. Today, newborn screening in Connecticut includes 65 medical conditions, including her daughter’s. “So these are lives that we actually end up saving. There are therapies for each of these rare disorders that are being screened and there is a big difference between children who has been treated from birth to go on to leave fairly normal lives (other than the fact that they have to take a medication for the rest of their lives) and ones who are not treated who end up like my daughter and they're wheelchair bound.”
Bennett ended the conversation noting that regardless if they are wheelchair bound or not, these children are living to adulthood and it places a huge burden on the states and the public health system that needs to be addressed at the local, state, and federal level.