Fact Sheet

Our Friend Max


07.07.15

A Pint-Sized Reminder Of Why We Need #Cures2015

This week the House of Representatives will consider H.R. 6, the 21st Century Cures Act. The Energy and Commerce Committee approved this legislation by a vote of 51-0 on May 21, 2015. That day, there was a very special guest in the audience.

Max anxiously watches the full committee vote on the 21st Century Cures Act

Max, a friend of anyone he meets, visited Capitol Hill on the historic day of the full committee’s unanimous approval of H.R. 6. His joy was contagious.

Max sits in the chairman’s seat with Chairman Upton (R-MI) and Rep. Diana DeGette (D-CO)

Max, his family, friends, and the RASopathies Network USA (of which his mother is the Vice President), have a very special interest in the 21st Century Cures Act and have supported the initiative throughout the past year and a half.

When Max was born his mother noticed he had wide spaced eyes and low set ears, and Max was quickly taken to the neonatal intensive care unit. He had low oxygen levels, pulmonary valve stenosis, atrial septal defects, failure to thrive, reflux, qualitative platelet defect, chiari malformation of the brain, and a tethered spinal cord. After a year and a half, Max was eventually diagnosed with Noonan Syndrome, a rare genetic condition caused by mutations in genes.

Max meets Rep. Gus Bilirakis (R-FL)

Rare conditions, such as Noonan Syndrome, are not as rare as they sound. One in ten Americans suffer from a rare disease and the amount of time and money it takes to find treatments for these is significant. There are many barriers throughout the process. The 21st Century Cures Act aims to remove these barriers and streamline the process of getting new cures and treatments to patients in a timely and cost-efficient manner.

The 21st Century Cures Act helps incentivize more research for rare conditions, gives more financial support to the National Institutes of Health and Food and Drug Administration, streamlines the drug and device development process, and, importantly, strengthens the patient voice in regulatory decisions.

Max chats with Rep. John Shimkus (R-IL) following the full committee vote

Max, like many other patients, made this legislation possible. He has remained positive, hopeful, and dedicated to advocating for 21st Century Cures. Max has been writing letters and meeting with policymakers to draw support throughout the process. On the day of the full committee vote, Max was there to meet with committee members and staff and to cheer on the legislation that brave patients like him helped inspire. Legislation that will help all patients, families, and loved ones in America. Legislation that will help our good friend Max.

View more photos from Max’s visit HERE.

Learn more about RASopathies HERE.

Learn more about H.R. 6 HERE.

###

Fact Sheet