Cure SMA Releases Statement on 21st Century Cures

ELK GROVE VILLAGE, Ill. - Jan. 28, 2015 - PRLog -- Cure SMA, the leading organization in the US dedicated to the treatment and cure of spinal muscular atrophy (SMA), has issued a statement commending the US House of Representatives Energy and Commerce Committee for their release of the 21st Century Cures discussion document.

An initiative created by the Energy and Commerce Committee, 21st Century Cures investigates how the government can help "accelerate the discovery, development, and delivery of promising new treatments and cures for patients."

This initiative is led by Representative Fred Upton, who also chairs the Energy and Commerce Committee. Chairman Upton was one of the honored guests at Cure SMA’s 2014 Hope on the Hill congressional dinner.

“We’re pleased to see Chairman Upton and the committee taking such a proactive stance on drug development and approval. Like over 90% of rare diseases, SMA doesn’t have an approved treatment. However, we’ve made huge strides in research, and we are closer than ever before to a treatment for SMA,” said Jill Jarecki, PhD, Cure SMA’s research director.

“When treatments for SMA are ready, we want to see them reach our community as quickly and safely as possible. This discussion document represents an important first step toward that goal. Further work will be needed to review and refine these proposals and—mostly importantly—to translate them into legislation that will ultimately be passed by the House and Senate, and signed into law.

“Along with many of our partners in the rare disease sector, we look forward to being a part of that process.”

An estimated 70% of known diseases qualify as rare diseases, including spinal muscular atrophy. Fewer than 10% of those have an approved treatment. The cost and length of the drug approval process, combined with a high failure rate, can make it difficult to improve those statistics.

After a series of hearings, meetings, and roundtables, the committee identified several areas for improvement, including incorporating patients into the regulatory process, aiding young scientists who may help develop innovative cures, streamlining the regulatory process, and modernizing clinical trials and medical product regulation.

This discussion document summarizes their findings and proposed legislative solutions. As the title would suggest, the goal is to generate discussion that can further refine these proposals. Once this phase is complete, the committee will introduce formal legislation to the House of Representatives.

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About Cure SMA

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person’s ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

Since 1984, we’ve directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children’s bodies, extend life, and lead to a cure.

We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we’ll do everything we can to support children and families affected by the disease.

Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.