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Patients Fighting Deadly, Rare Lung Disease Issue Urgent Plea for Access to First and Only Treatment Option
Tuesday, May 14, 2013
Earlier this year, Canadians diagnosed with idiopathic pulmonary fibrosis (IPF) were elated to hear that the first medication was finally available to help them fight this rare and deadly lung disease. Months later, these patients are surprised and dismayed to be facing barriers to accessing this
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TGen Children’s Research Funded by May 19 BBQ & Concert
Tuesday, May 14, 2013
Mitochondrial disease took the life of 5-year-old Mylee Grace of Phoenix, but a fundraiser in her memory for the Translational Genomics Research Institute (TGen) could change the outlook for other children in the futureA barbecue and live music concert is planned from 12-4 p.m. Sunday, May 19, at
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Patients Rally to Raise Awareness of Rare Disease
Monday, May 13, 2013
Nina Smith is one of the approximately 25 to 30 million Americans with a rare disease. She lives with hereditary angioedema (HAE), which causes painful, unpredictable and potentially life-threatening swelling attacks throughout her body, including her arms, legs, face, abdomen and upper airway.
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Two Best Friends, Ages 6 and 7, Raise $200,000 to Fight Rare Disease
Monday, May 13, 2013
There are only about 100 people in the U.S. like 7-year-old Jonah PournazarianHe suffers from a rare genetic and incurable disease called Glycogen Storage Disease Type 1B. Up until the 80s, most kids with his condition didn't survive past the age of twoBut his best buddy, Dylan Siegel, wanted his
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National Organization for Rare Disorders to Celebrate 30 Years of Achievement and Promote a Promising Future for the Rare Disease Community
Monday, May 13, 2013
The 30th Anniversary Gala of the National Organization for Rare Disorders (NORD), to be held Tuesday, May 14, 2013, will honor rare disease heroes, celebrate 30 years of progress through the Orphan Drug Act (ODA) and NORD, and promote a promising future for the rare disease communityThis seminal e
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Patients Rally to Raise Awareness of Rare Disease
Friday, May 10, 2013
Nina Smith is one of the approximately 25 to 30 million Americans with a rare disease. She lives with hereditary angioedema (HAE), which causes painful, unpredictable and potentially life-threatening swelling attacks throughout her body, including her arms, legs, face, abdomen and upper airway.
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Logan Is Always Smiling, but It's Really a Symptom of His Rare Genetic Disorder
Friday, May 10, 2013
But for Logan McKechnie's family, his constantly happy nature is a reminder of the difficulties that life holds for himLogan, from Ballywalter in Co Down, has Angelman Syndrome, a rare genetic disorder which causes severe learning difficulties and leaves him unable to speakOne of the side-effects
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Burg Family Battles Disease Together
Friday, May 10, 2013
It took numerous doctors almost three years to figure out what was causing Connor Laframboise's symptoms - visual impairment, severe respiratory infections, aspirating food and liquidsAt almost four years old, Connor is unable to walk or talk and now must be fed through a feeding tubeA London tea
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Former FDA Official Tim Cote Joins Emmaus Medical as Regulatory Advisor
Friday, May 10, 2013
TORRANCE, Calif. - Timothy Cote, M.D., MPH, former director of the Office of Orphan Product Development of the U.S. Food and Drug Administration, has joined Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine company, in an advisory capacity, the company announced today. Dr.
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Rotary Honors U.S. Government for Leadership on Polio Eradication
Thursday, May 9, 2013
WASHINGTON - Today, Rotary International will recognize five members of Congress as Polio Eradication Champions for their efforts to eliminate polio. The event, which will take place at the Capitol Building in Washington, D.C., honors U.S. leaders who have played a key role in the fight to The 20