On January 18, 2024, 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…
On December 5, 2023, the Rare Disease Legislative Advocates (RDLA) held an RDLA Caucus briefing. This briefing, held in collaboration with the Rare Disease Congressional Caucus, provided an update on…
Many helpful guides cover immunology which is the study of the immune system and how the body defends itself against disease. The immune system is comprised of cells, organs, small…
Happy holidays to the Vallecalle family, who are rejoicing in the fact their baby girl is cured of the rare disease, hyperinsulinism. Not only that, but they finally get to…
We can all say that our mothers gave us the gift of life. But how many can say that they gave the gift of life in return? One teenager in…
According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
Six years ago, Jonathan Kowalske's son Mac entered the world - and the family suddenly found themselves thrust into the rare disease community. Soon after birth, Kowalske and his…
Unfortunately, Jody McPherson, 47, a veteran firefighter with the Mesquite Fire Department, passed away after his battle with Creutzfeldt-Jakob disease (CJD). As reported by Selena Hernandez of CBS News Texas,…
Two emergency surgeries that began 48 hours after birth, meningitis, and over a month in the intensive care units of three Seattle hospitals prompted Layla Babayev’s desperate parents to enroll…
Four-year-old Emmett Doucette is currently fascinated by firemen, dinosaurs (he has a favorite T-rex toy!), and toy race cars. He’s known to wear a fun fireman outfit and he loves…
For months, 47-year-old Jane Lightfoot struggled with what she thought was a nasty ear infection. The spot behind her right ear ached; she was dizzy, couldn’t hear well, and the…
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,…
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