Trevor O'Sullivan Trevor O'Sullivan, who lives in Ireland, has cystic fibrosis (CF). He has undergone two liver transplants. He was the 42nd person in the country to have this surgery.…
Mark Cohen is a 28 year old lawyer who has a rare eye disease called choroideremia. Its genetic, and his grandpa, brother, and cousin were all diagnosed. He didn't receive…
Social distancing sucks for everyone. Even introverts (like myself) are becoming antsy. Similarly, the coronavirus is scary for everyone, even those less likely to be effected by serious illness. However,…
NICHD The NICHD is the The Eunice Kennedy Shriver National Institute of Child Health and Human Development, one of the National Institutes of Health (NIH). It is the center for…
Social Distancing Social distancing has been coined as the most surefire way to stop the spread of COVID-19 across the American populace. What is this phrase used to refer to?…
No one was given a prep course on how to handle quarantine, especially not with kids. Here are some suggestions to help you get through these unique times, while making…
Every day on the news we hear the pleas for more medical masks for doctors and nurses, more personal protective equipment, and more ventilators. We all know what masks are,…
In this time of cancellations, closures, and shutdowns, many companies and individuals have found unique and creative ways to shift their services online. Better yet, some have temporarily waved their…
What is Repurposing? Repurposing involves testing whether or not a compound approved of another condition may be capable of also treating an illness with no approved treatment option. It's advantageous,…
By now, everyone has heard of COVID-19. Information is constantly being updated surrounding the nature, symptoms, spread, and potential treatments for the illness. Despite this constant influx of information, there…
What is Homocystinuria? Classical homocystinuria, or HCU, is a rare metabolic condition which its caused by a deficiency of the CBS enzyme. It results in high levels of homocysteine in…
Relapsing polychondritis is a rare disease which causes damage to the cartilage, trachea, and lungs. It's an autoimmune condition meaning an overzealous immune response causes the body to attack its own…
A recent study, conducted my Melinda Kavanaugh from the University of Wisconsin, Milwaukee, examined the role of children who are acting as caregivers for those diagnosed with amyotrophic lateral sclerosis…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour…
Published in Social Science & Medicine, a recent study conducted by Drexel University has shed light on how, even in a world of evolving precision medicine, there is also merit in…
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…
It's easy to feel helpless right now. You're told to stay home and just wait it out. So you're stuck feeling isolated and angsty, knowing there's nothing you can personally…
Chronic granulomatous disease (CGD) is a rare form of immunodeficiency. The most common form of this disease is X-linked chronic granulomatous disease (X-CGD) which is caused by a mutated CYBB gene. This…
The Fighting HARD Foundation Rare diseases are difficult to face for a myriad of reasons. They can be financially burdensome, physically painful, and emotionally draining. Further, they can make patients…
The European Parliament has announced that they will be relaunching their "Network of Parliamentary Advocates for Rare Diseases." Its aim is to create a new policy framework which can improve…
Researchers all over the world are hard at work trying to develop both treatments for Covid-19 and vaccines for the illness. Professors of rheumatology at the University of Alabama, Randy…
Social distancing sucks in so many ways. I'll be the first to admit it. You can feel isolated, alone, stuck, scared. But, there are some upsides to this crazy situation.…
We know that older individuals and those who are immune compromised are more at risk for experiencing severe Covid-19. These include multiple sclerosis (MS) patients. Multiple Sclerosis is a rare…
Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…
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