At first, everything in Amberlyn Smith's pregnancy seemed to be going smoothly. When she was just over 29 weeks pregnant, her screening tests appeared to be normal. However, at…
Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT)…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
According to a story from bloomberg.com, the biopharmaceutical company Rallybio has announced two development programs for therapeutics intended to treat rare diseases. The two programs consist of RLBY212, which is…
Aljazeera recently published an article that highlights the difficulty several families have had waiting for a rare disease diagnosis. In the majority of cases, when the families finally received the…
A recent article in the journal Nature describes the first draft of human genome analysis and sequencing published in 2001 by the Human Genome Project international consortium. The consortium included…
Parker Boyer and his family know many of the obstacles that rare disease patients face; they've encountered many of them since Parker's birth. In fact, they dealt with a number…
When a patient is told that they have a life-threatening rare disease and then told that there is no cure, every effort is made to attend to their physical needs.…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
Together, Takeda Pharmaceuticals and the Children's National Hospital have created a program that aims to create and improve a standard of care for rare disease patients. As these patients often…
A recent study has documented the differences between two rare forms of childhood brain inflammation. The two similarly presenting conditions are often confused for one another. A better understanding of…
During the National Institutes of Health (NIH) Rare Disease Day at NIH event, a panel was held titled "From Kitchen Tables to Changing Paradigms — Advocacy as a Driving Force…
Crispr is no longer a buzzword. According to a recent article in The Guardian, it has become the ‘molecular scissors’ that will enable scientists to rewrite our genes or as often said,…
As part of ongoing events surrounding Rare Disease Day, which is officially recognized on the last day of the month of February, Patient Worthy attended sessions of the 8th Annual…
Ray Jordan and Cyndi Segroves were born thousands of miles away from one another; in fact, they grew up in different countries. Cyndi is from the US and Ray is…
During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative…
Biotechnology company Stealth Biotherapeutics ("Stealth") has made the company's mission to develop and commercialize treatments for patients with diseases characterized by mitochondrial dysfunction. One leading treatment in the company's…
According to a story from Sano Genetics, Sano Genetics, BenevolentAI, C4X Discovery, and Patients Know Best expect only the most from their novel collaboration. Together, they are pooling top expertise…
Research by scientists at Cornell University and Weill Cornell centered around the endothelial cells that form a barrier between tissues and vessels. They control the flow of fluid and substances…
As the pandemic rages on, doctors across the country are beginning to notice a trend. A number of children who have contracted COVID-19 have also been impacted by a rare…
If you’re a caregiver for a loved one with a rare disease, it’s not presumptive to assume you work tirelessly managing your loved one's busy appointment schedule, daily stretch routine,…
Raghav is a two-year-old boy born with an ultra-rare disease caused by a mutated GPX4 gene. He and eight other children are the only ones in the world who have…
Arteriovenous malformation (AVM) is a rare disease that causes blood vessels to clump together within the skull. It can lead to aneurisms, strokes, or early death. However, surgery is possible…
The last day in the month of February is recognized each year around the world as Rare Disease Day. The day serves primarily as a moment of spreading awareness about…
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