Search

Washington, D.C., December 7, 2016 — Today, the United States Senate overwhelmingly approved the 21st Century Cures Act, a game-changer for medical innovation, by a bipartisan vote of 94-5. The President is expected to sign the bill into law later this week.

“Passage of the 21st Century Cures Act is the culmination of several years of hard work and advocacy by many rare disease patient advocates and patient advocacy organizations,” said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), which has supported the bill. “The 21st Century Cures Act is a landmark medical innovation package that has the potential to help nearly every American family, including the 1 in 10 Americans with rare diseases who desperately need treatments and cures.”

The bill includes many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients, including:
  • An extension of the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes the development of new therapies to help the more than 15 million children with rare diseases;
  • Streamlining of U.S. Food and Drug Administration (FDA) review of genetically targeted and protein variant therapies for rare diseases;
  • Creation of funds in the amount of $4.8 billion over 10 years for the National Institutes of Health (NIH) to fund the Precision Medicine Initiative, BRAIN Initiative, and the Cancer Moonshot;
  • Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulatory review.

NORD has participated in the 21st Century Cures Initiative since its inception in the winter of 2014 by submitting policy proposals for the initiative and testifying in hearings before the House Energy and Commerce Committee to provide the rare disease patient perspective. The organization has also published multiple position statements, led and joined coalition letters, and organized advocacy alerts to rally patients and advocates.

Added Saltonstall, “We were proud to help lead the charge to reauthorize the Rare Pediatric Disease Priority Review Voucher program, help author the provision to streamline review of targeted therapies for rare diseases, bring attention to the importance of NIH funding, and advocate for greater transparency of expanded access policies.

“After several years, we are excited to celebrate the Congressional enactment of the 21st Century Cures Act. Once again we wish to convey our deep gratitude. This bill would have never passed without the hard work of patient advocates over the past several years. There is still work to be done, but this is a major victory. Progress has been made, and with the ongoing effort of patient advocates working together for a better future, progress will continue,” Saltonstall concluded.

There are an estimated 7,000 rare diseases, which are defined as a disease affecting 200,000 or fewer Americans. Of the nearly 1 in 10 people with a rare disease, more than half are children. Nearly 95 percent of rare diseases have no treatment and the majority of these diseases are life-altering or life-limiting.

The National Organization for Rare Disorders (NORD) is a 501(c)(3) independent charity and the leading independent advocacy organization representing all patients and families affected by rare diseases.

Read the statement at rarediseases.org

Melvin Carruth

This letter is a tribute to my brother Melvin Carruth! We believe he is one of the oldest living African Americans with Maple Syrup Urine Disease.

Read More

NBS-MSUD Connect: Your One-Stop Shop For MSUD Resources

NBS-MSUD Connect was launched as part of the Newborn Screening Connect patient registry (NBS Connect) in 2013 through a partnership between the Department of Human Genetics at Emory University, the Maple Syrup Urine Disease (MSUD) Family Support Group and other key stakeholders.

Read More

Cambrooke Therapeutics

Cambrooke Therapeutics continues to expand its line of delicious and nutritious low protein foods to help improve the lives of individuals with Inborn Errors of Metabolism such as MSUD.

Read More

Updates To Nutrition Management Guidelines

The Nutrition Management Guideline for MSUD was first published in 2014. Since that time, there have been reports of new research and experiences that have prompted updates of the guideline.

Read More

Emory Metabolic Camp 2018 Announcement

Join us June 18-23, 2018 for the 24th Annual Metabolic Camp at Emory University in Atlanta, GA!

Read More

From The Chairman’s Desk

As I sit here reflecting on how much the care and treatment of MSUD has changed over the years, my mind goes back to 1978 when our son Keith was born.

Read More

MSUD Advocacy Report

Medical Nutrition Equity Act

The Medical Nutrition Equity Act (MNEA) would require all private insurance plans (state regulated or self-insured/self-funded) and federal health programs, including Children’s Health Insurance Program, Tricare, Medicaid, Medicare, and Federal Employee Health Benefit Plans, to provide coverage for formula and low-protein foods for all children and adults with MSUD.

Read More

The MSUD Family Support Group Is Excited To Announce Their Participation In The Million Dollar Bike Ride

The MSUD Family Support Group is excited to announce their participation in The Million Dollar Bike Ride

Read More

19th Biennial MSUD Symposium

I can’t believe that almost two years have passed and it is time for another MSUD Symposium! I am especially excited about this conference because I’ve built in extra time for social interaction.

Read More

Change the lives

of MSUD adults and children

Subscribe to our mailing list

Signup To Our Newsletter Signup with your email address to receive news and updates