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Father to Embark on 220-mile Bike Ride for Sisters With Genetic Disorder
Tuesday, May 21, 2013
A FATHER-of-two from Thornbury will tackle a strenuous 220-mile cycle across rocky terrain for two sisters whose rare genetic disorder leads them to jerk uncontrollablyPhil Lucker, 36, will take on the Escape the Dragon challenge, riding from Caernarfon in Wales back to Thornbury in three days bet
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Blood Donations Give Life to Youngsters Harper and Banjo, Who Have a Rare Blood Disorder
Tuesday, May 21, 2013
The Evanston Park siblings suffer from autoimmune lymphoproliferative syndrome, a rare chronic blood disorder that causes the immune system to attack red blood cells and the spleen to swellTo survive, Harper and Banjo rely on immunosuppressants and undergo regular blood transfusionsTheir mother
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Boy With Rare Disease Inspires Joy in Many
Tuesday, May 21, 2013
Many in Waco are finding inspiration in a very unexpected wayDespite his intense struggle with a rare disease, 10-year old Jaylon is spreading joy to all around himTo anyone who's met him, he's simply known as "J-Bug"You meet him, and you love him," said J-Bug's mom, Pepper JonesBut when J-Bug
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Patient Involvement in Drug Development Recognized as Critically Important at GTC Bio
Tuesday, May 21, 2013
Patient involvement across the drug development lifecycle was a central theme of the presentations and discussions at the GTC Bio Orphan Drugs Research & Commercialization Conference (held in Boston, MA on May 8-10, 2013Speakers recognized that the pharmaceutical industry has traditionally engaged
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LYSOGENE Announces Awareness Activities Dedicated to the “International MPS Awareness Day 2013”
Tuesday, May 21, 2013
May 15, marks the International MPS Awareness Day. It honours MPS children and their family member’s that courageously live with this diseaseIn order to support the International MPS Awareness Day 2013, LYSOGENE set up different activities targeted to patients and caregivers, medical professiona
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Connor’s House’ Helps Families Affected by Rare Childhood Disease
Friday, May 17, 2013
Some of them are so rare that they affect only one child in a millionAll of them have names the average person couldn't and wouldn't have reason to pronounceThey're all insidious. They are all incredibly rare. And they are all, sadly, almost always fatalWhen families are affected by these rare d
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RARE DISEASE: Story Co. Sheriff Taken Ill
Friday, May 17, 2013
The Story County Sheriff has been diagnosed with a Guillain-Barre syndrome according to a Story County Democrats emailSheriff Paul Fitzgerald was hospitalized in Ames last Wednesday with the disorderIt’s an uncommon illness where a person’s immune system attacks the nerves. Weakness and tingl
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NPS Pharmaceuticals Recognized by National Organization for Rare Disorders for Gattex® As an Innovative Treatment for Patients With Short Bowel Syndrome
Friday, May 17, 2013
NPS Pharmaceuticals, Inc., a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, announced that the company was honored by the National Organization for Rare Disorders (NORD) for the FDA approval of Gattex® (teduglutide [
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Synageva BioPharma™ Joins the International MPS Network in Support of MPS Awareness Day 2013
Friday, May 17, 2013
Synageva BioPharma Corp. (Synageva), a biopharmaceutical company developing therapeutic products for rare diseases, joins the International MPS Network and others around the world in observing MPS Awareness Day 2013. During MPS Awareness day, patient organizations, industry, and other participants
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Bridport Family Walk to Help Grandson With Rare Genetic Disease
Friday, May 17, 2013
THE Hiller family received news just before Christmas that three-year-old Joe had a rare genetic disease that is likely to end his young life by his tenth birthdayIt was undoubtedly the family’s worse Christmas, said grandad Terry from Mead Fields in BridportJoe is one of only 26 children in th