Rare Disease Day: Here's why quality research matters

In honor of Rare Disease Day today (Feb. 28), U.S. Rep. Gus Bilirakis, R-Tarpon Springs, introduced the Orphan Product Extensions Now Accelerating Cures and Treatments Act to help open the door to potentially hundreds of new treatments for rare diseases.

"Rare disease patient communities need to come together on a common platform".

With most rare diseases having no cure or even effective treatment available, living with or having someone who has a rare disease can be extremely traumatic both physically and mentally. Together with members of the rare disease community, ProQR Therapeutics N.V., another biopharmaceutical company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases has also joined the said endeavor. Thanks to this celebrity-fueled craze, this "rare disease" was shoved under the public's eye and received $115 million dollars in funding donations in just 30 days, helping provide a major genetic breakthrough towards understanding the disease.

"Lack of awareness and scientific knowledge often leads to misdiagnosis or late diagnosis and delayed treatment".

More than 150 rare disease patient advocacy groups support the OPEN Act, including the EveryLife Foundation, the RASopathies Network, the Pediatric Cancer Foundation, and more.

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ISCR said out of the 7,000 known rare diseases today, majority are "progressive, life-threatening, and chronically" debilitating conditions. As part of the campaign, which will run until March 6, the agency has wrapped the inside and outside of a train vehicle on the Massachusetts Bay Transportation Authority (MBTA) Red Line with facts about rare diseases and zebra stripe branding.

Rare Disease Day takes place on the last day of February each year.

About the National Organization for Rare Disorders (NORD)® The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases.

The Indian Society for Clinical Research (ISCR) is an association of clinical research professionals that aims to build awareness of clinical research as a specialty in India and to facilitate its growth in the country while helping to evolve the highest standards of quality and ethics.

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