SOURCE: Jonah's Just Begun

Jonah's Just Begun

January 18, 2012 10:00 ET

Jonah's Just Begun Supports Bipartisan Legislation That Incentivizes Research & Development for Rare Diseases and Accelerates Drug Approval Process

NEW YORK, NY--(Marketwire - Jan 18, 2012) - Jonah's Just Begun (JJB), a non-profit organization dedicated to finding a cure for Sanfilippo Syndrome, a rare, fatal disease typically diagnosed in preschool years -- today signed the Letter of Support for The Unlocking Lifesaving Treatments for Rare Diseases Act of 2012 (ULTRA). ULTRA was introduced by two senior members of the House Energy and Commerce Committee, U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY) to incentivize and accelerate the development of treatments for very rare diseases.

Despite the success of the Orphan Drug Act signed 25 years ago there are very few treatments for rare diseases that affect very small patient populations. According to the U.S. Food & Drug Administration approximately 30 million Americans have a rare disease. In the U.S. the known Sanfilippo patient population is 18.

"My child was born with a fatal, rare disease and despite my crusade he may die because of the way drug development currently works in this country," said Jill Wood, founder and CFO of Jonah's Just Begun. "This legislation gives my son Jonah a chance and so I ask all the members of Congress, what would you do if this were your child?"

Sanfilippo Syndrome is a genetic metabolism disorder that prohibits the proper breakdown of sugar molecules in the body. The syndrome belongs to a group of diseases called mucopolysaccharidoses (MPS). Currently newborn screening is not offered for Sanfilippo Syndrome and many other rare diseases. Symptoms of Sanfilippo usually appear after one year of life. Sanfilippo causes severe neurological damage. Brain damage is followed by a series of catastrophic events to the body's bones and organs. Death usually occurs by the second decade of life.

"JJB's team of scientists expect a treatment to be ready for clinical trials in a few years," said Alexei Pshezhetsky, professor of pediatrics, University of Montreal, Medical Genetics. "Unless there is the possibility for a fast-track approval process, more children will suffer and die."

Jonah's Just Begun -- Foundation to Cure Sanfilippo Inc. is a family-run organization. With the help of JJB's sister organizations and medical board the stage has been set for finding a cure. An international research team is currently collaborating on various treatment options, the first ever type C mouse model has been created and initial work has begun for our Natural History Study. JJB's team of scientists estimate a treatment will be ready for FDA review for clinical trials in a few years.

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