Rare Disease Day shines a light on diseases, community, initiatives
Patient Advocacy Initiative staff speak on Notre Dame’s Rare Disease Day event and impact for the rare disease community.
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.
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: MNH chief calls for enhanced awareness initiative
MUHIMBILI National Hospital (MNH) executive director, Prof Mohamed Janabi has emphasised the need for greater public awareness on rare diseases to help communities understand the challenges faced by patients.
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.
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.
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
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Rare Disease Day: Richmond mother turns grief into advocacy after losing son to rare disease
Donna Reynolds of Richmond knows all too well the devastating toll that a rare disease can take, not just on an individual, ...
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.
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5 Rare Diseases That Now Have Their First FDA-Approved Treatments
Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...
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 ...
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Hospital Kuala Lumpur & Rare Disease Patient Organizations Nationwide Celebrate Rare Disease Day 2025
For the first time, Hospital Kuala Lumpur (HKL) is proudly hosting the annual Rare Disease Day 2025 celebration to raise ...
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 ...