Family finds rare disease diagnosis after 14-year search
MADISON, Wis. (WIFR) - For 14 years, Treyson Wallace’s family searched for answers. Born in 2009, Treyson failed a newborn hearing test, missed developmental milestones and struggled to walk and learn. Despite countless doctor visits and tests, no one could explain why - until now.
Rare Disease Day 2025: Know Date, History, Significance And More
Rare Disease Day is observed annually on the last day of February to raise awareness for the 300 million people worldwide living with a rare disease, their families, and carers. The day aims to improve the lives of those living with rare diseases by promoting equity in social opportunity,
Arrowhead, Zevra fire up campaigns for Rare Disease Day 2025
Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won FDA approval for a Niemann-Pick disease type C (NPC) treatment, Miplyffa, last year and recently agreed to sell a priority review voucher for $150 million to fund the launch and other work.
Rare Disease Day: seven drugs awaiting approval in 2025
As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in 2025.
Recognizing Rare Disease Day raises awareness for undiagnosed patients
This last week of February marks Rare Disease Day. This day aims to raise awareness of rare diseases and improve access to treatment.
What to know about the rarest day of the year on Feb. 28 or Rare Disease Day
Did you know that one out of every ten Americans has a rare disease? That's why Rare Disease Day is so important to so many people.
Rare Disease Day highlights awareness, research efforts happening in Roanoke and New River Valley
Rare Disease Day, observed on Feb. 28, brings attention to uncommon diseases affecting populations of 200,000 or fewer. In Roanoke, the Wells Fargo Tower will be illuminated in pink, green, purple, and blue—the colors of Rare Disease Day—thanks to a collaboration between The Fralin Biomedical Research Institute at VTC and Wells Fargo Tower.
Local family of boy with rare disease raises awareness, spreads hope
Feb. 28 is Rare Disease Day and Channel 9 is spotlighting a local family working to shed light on rare diseases all year long.
Rare Disease Day 2025: Raising Awareness On Stem Cell Breakthroughs
Rare diseases affect multiple organ systems and approximately 50–75% of them affect children. Nearly 80% of all rare diseases are estimated to have an underlying genetic aetiology. The Indian Council of Medical
Group at new Mount Sinai center aims to provide diagnosis to those living with rare diseases
In observance of Rare Disease Day, a new effort is underway to finally provide answers to the estimated 300 million people ...
After A Five-Tool Career, Bernie Williams Steps To The Plate To Take On Rare Disease Day
Williams, who has performed the National Anthem at baseball stadiums as part of Boehringer Ingelheim’s Breathless campaign, ...
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As Virginia Tech honors Rare Disease Day, researchers question the future of long-term funding
Roanoke's Wells Fargo Tower is lit up Friday night to bring attention to illnesses that impact one in every ten Americans.
World Rare Disease Day 2025: Apollo Navi Mumbai Saves 4-Year-Old With Chediak-Higashi Syndrome Via Life-Saving Bone Marrow Transplant
On World Rare Disease Day, Apollo Hospital Navi Mumbai highlighted a rare medical success story, treating a four-year-old ...
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'It’s As if Aiden Knows He Won’t Be Here Forever'—One Mother's Fight to Help Her Son Battle His Rare Disease
On Rare Disease Day, it's important to understand the battles families face to get the help for their kids, and how you can ...
Avidity Biosciences Honors Rare Disease Day® and Global Efforts to Support People and Families Impacted by Rare Neuromuscular Diseases
Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...