A recent study published in the American Journal of Gastroenterology has demonstrated that people diagnosed with eosinophilic esophagitis (EoE) are at a higher risk for anxiety, mood disorders, as well as ADHD. With this…
Fast Track designation is designed to facilitate the development of, and expedite the review of, drugs intended to treat serious conditions and fill unmet patient needs. This designation comes alongside…
Drug development is a long, expensive, and difficult process. Not every treatment being created makes it to the end of the clinical trial process, much less through FDA approval. Takeda…
Her brother, her sister, and her father - all people that Salima Mohip lost before they turned 43. Each of them passed away from heart attacks, a consequence of their…
According to a recent article, 17-year-old Diego Ramirez was able to compete in the Flying Pig Marathon despite his Duchenne muscular dystrophy diagnosis. Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy…
Fundraising is extremely important when it comes to rare disease research, and recently a community came together to help a little boy living with Sanfilippo syndrome. Simon is 5 years…
It’s no secret that teachers can make a huge impact on the students in their classrooms. And that’s just what Judy Griffin did for her student Ashley. When Judy started…
In the past, researchers have observed a relationship between hemophilia and bone-related issues. However, while this observation is helpful, there have been few studies examining the prevalence of bone problems…
The WHO’s Emergency Response System Has Been Activated. Just last month, a World Health Organization (WHO) representative assured VICE World News that the WHO is taking this global outbreak very…
According to a recent article, a man in Maine has died after being bit by a tick and contracting the rare Powassan virus from the bite. Powassan Virus Powassan virus…
When a patient or caregiver speaks of immunotherapy it is often with a more confident tone than when they are speaking about chemotherapy. However, the odds of a patient going…
ITP, or immune thrombocytopenia, is a platelet disorder where the immune system destroys its own platelets by way of destructive antibodies. Platelets are very small blood cells that originate in…
In the United States, the FDA grants Breakthrough Therapy designation to drugs intended to treat serious and life-threatening illnesses, and/or drugs which show significant improvement over available therapies. After receiving…
Jörg Richter is no stranger to difficult bike rides. In fact, shares KRDO, Richter has ridden his bike across the United States - from coast to coast - seven times…
When David and Melissa Wickham found out that they were expecting, the pair was thrilled. They had already been together for years – it took seven to conceive their daughter,…
Happy Friday! This week, we have details on an investigational compound for beta thalassemia. Also, we break down the details of a new study that examines psychiatric illness rates among…
We’ve reached the month of May, and you know what that means – it is Guillain-Barre syndrome (GBS) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Awareness Month! During this month, the…
Imagine the feeling of working for years with the goal of saving children who have congenital athymia. Then thirty years later the FDA approves a regenerative medicine based on your…
Alzheimer’s disease is the primary cause of the progressive decline of cognitive function in the elderly. It is characterized by underlying complications in the central nervous system (CNS). To date,…
LPCN 1144 for nonalcoholic steatohepatitis (NASH) has already demonstrated positive results in the LiFT trial, and it is now showing more exciting results in its open-label extension (OLE) trial. Want…
The joy of watching your child grow and meet developmental milestones is not always granted to some parents. Clare’s mother, Allison, described her emotions to CHOP after noticing that Clare…
Because of their rarity, rare diseases often aren't considered or represented in the government. But state by state, we are slowly gaining representation within the legal system. This is being…
For over thirty years, researchers at the University of Pittsburgh (UPMC) have been observing cases of limited scleroderma, a connective tissue disorder that causes thick hard patches of skin. Scleroderma…
A recent article highlighted the struggles of a man in South Yorkshire who has lost multiple jobs due to his diagnosis of cyclical vomiting syndrome (CVS). Cyclical Vomiting Syndrome (CVS)…
How does our body fight infections? After we get infected with a serious illness, in what ways does our immune system prepare? Understanding these processes could help hone treatments and…
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