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Mashup Score: 10
A brother and sister diagnosed with the same rare disease had very different outcomes because of a simple test. News4’s Shawn Yancy spoke with a Virginia mother who’s using her voice to raise awareness and give hope to others.
Source: www.nbcwashington.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0
The FDA approval of pozelimab (Veopoz) makes it the first and only treatment indicated for children and adults with CHAPLE disease.
Source: www.hcplive.comCategories: General Medicine News, General NewsTweet
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Mashup Score: 3Rare Disease Day brings attention to a puzzle lacking pieces | Emory University | Atlanta GA - 2 month(s) ago
Five-year-old Scarlett Alonzo has arginase deficiency, a rare metabolic disorder. Read her story and how she was part of a study testing an enzyme replacement therapy.
Source: news.emory.eduCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 11Many parents of kids with rare diseases fight for research — even if it’s too late for their child - 2 month(s) ago
“We’re still participating in this research even though our son passed away, and that still adds meaning to his life.”
Source: www.today.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 5Rare Disease Day: Moderna Gets Back to Its Roots - 2 month(s) ago
Even before its RNA vaccine for COVID-19 made Moderna famous, the company’s founders had a vision for treating rare diseases.
Source: www.genengnews.comCategories: General Medicine News, Future of MedicineTweet-
Rare Disease Day: Moderna Gets Back to Its Roots After being diverted by the SARS-CoV-2 pandemic to develop and manufacture COVID-19 vaccines, the mRNA therapeutics company has refocused on advancing #raredisease treatments through the clinic. Learn more: https://t.co/j42OaokwXG https://t.co/Cm26DDs1qW
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Mashup Score: 6IU's Undiagnosed Rare Disease Clinic helps girl with rare disease find answers after 16 years of uncertainty - 2 month(s) ago
IU School of Medicine’s Undiagnosed Rare Disease Clinic (UDRC) is helping families in Central Indiana uncover the mysteries behind their unknown genetic disorders.
Source: www.wrtv.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 32White House Rare Disease Forum - 2 month(s) ago
The White House
Source: www.youtube.comCategories: General Medicine News, NeurologyTweet
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Mashup Score: 4Turning to Patients to Help Drive Rare Disease Drug Development - 2 month(s) ago
Patient advocacy groups aided in the development of the very first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia.
Source: www.biospace.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 6Brewing up awareness for rare diseases - 2 month(s) ago
MUSKEGO — Mac Kowalske of Muskego is an active 6-year-old boy. The little athlete is a busy youngster with balancing school and playing on a hockey team for people with
Source: www.gmtoday.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 16
Welcome! You are invited to join a meeting: NORD Policy & Advocacy Town Hall. After registering, you will receive a confirmation email about joining the meeting.
Source: rarediseases.zoom.usCategories: General Medicine News, Rare DiseaseTweet
Check out this video featuring our Virginia @RareAction Ambassador and rare mom, Jana Monaco, and Dr. Deb Regier, Director of the Rare Disease Institute at @ChildrensNatl, one of 40 NORD #RareDisease Centers of Excellence: https://t.co/ah3S0mFw3d