This year, the National Organization for Rare Disorders (NORD) held its annual Breakthrough Summit from October 16-17 in Washington, D.C. During the Breakthrough Summit, NORD celebrated its 40th anniversary and…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
The goal of providing precision techniques to cancer care is becoming increasingly complicated. According to a recent article in Inside Precision Medicine, the cause may be attributed to the…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
As shared by MedScape, the U.S. FDA recently approved the first treatment for people living with congenital thrombotic thrombocytopenic purpura (TTP). Its name? Adzynma. In the past, people with TTP…
When 38-year-old Shereen-Fay Griffin went to the local swimming pool, she was hoping for some fun and relaxation. Instead, she contracted a rare but serious infection that left her blind…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Give the gift of life this holiday season and consider donating your bone marrow to BeTheMatch. Each year, over 18,000 people are diagnosed with a life-threatening blood condition that could…
An individual has a good chance of recovering from an injury to his or her legs or arms, but it is not always the case when the injury involves…
When it comes to rare and chronic conditions, awareness is important. Oftentimes, disease-specific information remains within the community; many others are informed about the impact of these conditions. Awareness not…
In a recent news release, biotechnology company InflaRx N.V. announced that the first participant had been dosed with vilobelimab in a Phase 3 clinical trial. Within the trial, researchers are…
26.2 miles. 5 boroughs. And a deep feeling of fulfillment when she finally crossed the finish line. When Holly Griffin first decided to run the New York Marathon, she was…
If Wave Life Sciences’ recently submitted trial application for WVE-006 is approved, it will become the first RNA editing treatment in a clinical development setting. Wave’s CEO, Paul Bolno,…
When Brittany Miller welcomed her daughter Brooke into the world, she had no idea what the next few months would bring. At just a few weeks old, Brooke began having…
Shortly after Laura Colby was born, she was diagnosed with a rare metabolic disorder known as maple syrup urine disease (MSUD). Normally, our body has an enzyme complex that breaks…
When Tucker Krause was a young boy, his family noticed that he frequently got sick. More than frequently, even. In fact, when his mom really thinks about it, Tucker was…
42o words (source - 2% match) vs. 479 words (mine - 1% match) Have you ever had a lingering itch? An itch that just wouldn't go away, no matter what…
On October 24, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
The xenotransplant involved transplanting Yucatan miniature pig kidneys into macaque monkeys with 69 genetic modifications. One monkey survived for over two years, offering hope to the team of Harvard…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Life is ever evolving so it is important to remember that the storms in our lives do not always last forever. When I developed dystonia in 2001, I lost everything I was…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Granite, marble, porcelain, quartz—all materials leveraged in the development of beautiful, durable countertops. When people are choosing what countertop material to use in their homes, they often consider factors like…
One of the most successful healthcare services benefiting the public is newborn screening. Unfortunately, this life-saving health initiative is not provided for all rare diseases in some U.S. states. As…
Written by Sara Tompkins NOTE: The perspectives voiced in this article are solely those of the author. The development of drugs for rare diseases has always been a complex and…
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