Pharmac Seeks Feedback on New Rare Disease Fund

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Pharmac is seeking public feedback on a new funding approach specifically geared for high-cost medicines for rare disorders. The use of a contestable fund, worth an estimated $5 million a year, will be tested this year, Pharmac chief executive Steffan Crausaz says. Suppliers will compete […]

GLOBAL GENES™ Launches “RARE Toolkits” as Premier Educational Resource for the Rare Disease Community

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Series of RARE Disease Toolkits Enable Advocates, Patients and Caregivers to Empower Themselves and Inspire Others With 1 in 10 Americans living with rare diseases, the need for access to powerful, actionable information has never been greater. Global Genes™ is proud to launch RARE Toolkits, […]

New Educational Toolkits from Global Genes Demystify Rare Community’s Biggest Problems

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Ask any parent to whip out their highlighted copy of the latest edition of Parenting a Child with a Life-Limiting Illness and you’ll be met by blank stares–or at least, you would have–until now. With 1 in 10 Americans living with rare diseases, the need […]

Undiagnosed: Medical Refugees: San Diego Event and Silent Auction

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April 25th is Undiagnosed Children’s Day! In raising awareness of the millions of disabled children living without a diagnosis, please help support a documentary currently in production that captures the arduous journey of patients and their families who are suffering without a diagnosis. On Friday […]

Support Group Meeting Challenges for Rare Diseases

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Pompe disease is so rare that few doctors in the world knew about it. What is even fewer are the treatment options available considering this genetic disorder is not at the top of the agenda of many drug research companies. This was the situation that […]

Philadelphia Bike Ride in May Will Raise Money for Rare Disease

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By Jeanne Leong Studying and finding treatments for rare diseases such as hemophilia and cystic fibrosis require hundreds of thousands of dollars in funding, as researchers at the Penn Center for Orphan Disease Research and Therapy in the Perelman School of Medicine know all too well. Orphan diseases—which total […]

Meghan Talks Bravery When it Comes to Cowden Disease

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At just two weeks post-op from a complete thyroidectomy, Cowden’s patient Meghan Ortega addressed her school during a movie night screening of Brave. In honor of World Rare Disease Day, Meghan bravely took the podium to discuss what life is like for kids with rare […]