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SOURCE Agility Clinical
Annual Rare Disease Day event to benefit rare/orphan disease research
CARLSBAD, Calif., Jan. 28, 2014 /PRNewswire/ -- Agility Clinical, Inc., a specialty consulting and contract research organization, and BIOCOM will host "Overcoming Challenges in Orphan Product Development", a special event in support of Rare Disease Day 2014 on February 27 at the Sanford Consortium for Regenerative Medicine in La Jolla, CA. The event will feature speakers who are experts in orphan product development as well as a silent auction to benefit HIBM Research Group (HRG). HRG is a non-profit organization dedicated to finding a treatment for Hereditary Inclusion Body Myopathies (HIBM).
"We are thrilled to help raise awareness for orphan drug development and to help support efforts to find a treatment for HIBM, a group of rare, muscle-wasting disorders," says Ellen Morgan, CEO of Agility. "This year, our speakers include brothers who are physicians, fathers, HIBM patients, advocates and researchers of the disease themselves, and who provide a truly unique perspective on the challenges of both living with a rare disease and finding treatment options."
Agility Clinical helps pre-clinical and clinical stage companies develop drugs and devices by providing strategic and operational consulting support, in addition to traditional CRO services, and specializes in orphan drug development.
"Our goal at Agility is to help biopharmaceutical companies overcome challenges in bringing treatments to patients suffering from rare diseases," says Marilyn Carlson, D.M.D., M.D., RAC, Vice President of Medical, Regulatory and Scientific Affairs at Agility.
The term "orphan product" refers to a pharmaceutical drug or a device developed specifically to treat a rare disease. Rare diseases affect one in 10 Americans, 30 million people in the United States, and 300 million people globally. Of the approximately 7,000 identified rare diseases, less than 500 have an FDA-approved treatment available. Many have genetic origins and affect young adults and children. It is estimated that more than 50% of those affected by rare diseases are children, making rare diseases the most deadly and debilitating diseases for children worldwide. For patients, getting a diagnosis can become a quest. Too often, patients are confronted with a lack of treatment for their condition. Biopharmaceutical companies working on bringing orphan treatments to patients face many challenges as well, from regulatory strategy to planning and conducting successful clinical studies.
Agility Clinical was founded in April 2012, with an experienced executive management team whose mission is to help small and virtual pre-clinical and clinical-stage companies develop drugs and devices, particularly in the orphan product category. In addition to consulting, Agility provides customized full clinical trial services including project management, clinical operations, data management, biostatistics, medical, regulatory, and scientific affairs and medical writing. The Agility team has a true appreciation for the challenges its clients face in obtaining the expertise they need. The firm's senior staff volunteers time with its partner, the Public Health Alliance for Clinical Trials (PHACT), the first non-profit CRO dedicated to conducting clinical trials for products to treat diseases that affect the poorest people in developing countries. Agility has established operations in Carlsbad, CA. For more information, visit www.agility-clinical.com and http://www.phactcro.org. For more information on the benefit for HRG, please visit www.agilityrarediseasefund.org
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