Rare disease research can be difficult to conduct, with one of the major contributing factors being funding. Research is expensive, and I mean really expensive. That's why grant funding and other…
When it comes to rare diseases, many doctors simply just don't know the proper way to diagnose, monitor, and treat them. It's difficult to know how to respond to every…
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…
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…
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…
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…
Christine Harrison, a grandmother from Poringland, has recently passed away due to progressive supranuclear palsy (PSP). She led a full, rewarding life filled with family and love. She's survived by…
Aiden Collyar, a nine-year-old from New Hampshire, was recently presented with a donation that would allow him to do something he loves: play sports. After he was diagnosed with the…
The process of an organ transplant is a long one, and I'm not just talking about the procedure itself. For many patients, the majority of the time is spent waiting…
It's time to raise awareness for and celebrate the patient population that lives with eosinophilic disorders. From May 15th until the 21st, the American Partnership for Eosinophilic Disorders (APFED) is…
The diagnosis of rare diseases can be difficult; they may share symptoms with other disorders, many are not well known, and many don't have specific diagnostic processes. Ankylosing spondylitis acts…
Raising awareness is extremely important when it comes to rare diseases. After all, rarity typically results in minimal public and medical awareness. That's why patient communities and other dedicated individuals…
A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
Rhabdomyosarcoma is a rare form of cancer that affects children. It was recently studied at St. Jude Children's Research Hospital, where a team of medical professionals examined cells that persist…
Lennox-Gastaut syndrome (LGS) is a rare, severe form of epilepsy that is often resistant to anti-seizure medications. This means that patients still experience epileptic episodes despite treatment. Because of this…
It is no secret that there is an inequity when it comes to race and medical research. More often than not, research does not take all races and ethnicities into…
Acromegaly and gigantism are two disorders that share a main characteristic: abnormal growth. Because of this similarity, many people confuse one for the other. However, these are two distinct, separate…
Compiling data, especially rare disease data, can be extremely helpful for research purposes and for doctors treating patients with that condition. Recently, the International Gaucher Alliance and Cerner Enviza have teamed…
A recent study published in Science Translational Medicine has investigated a possible factor of the development of fetuses born to mothers with neuromyelitis optica spectrum disorder (NMOSD). Specifically, it looked…
A parent never wants their child to endure a medical issue, much less one as serious as bacterial meningitis. Unfortunately, Jay Aston - a singer from Bucks Fizz - has…
Understanding which patients are at a higher risk of poorer outcomes is essential to provide them with the best treatment. That's why so many researchers focus on signs or markers…
Many father-son duos are close, but this pairing - reported on in an article from CBS58 - shares a special bond that was strengthen by the shared experience of a…
Many drugs, whether they are indicated for common or rare diseases, come at very high prices. Zynteglo, an investigational gene therapy for beta thalassemia, is most likely to stick to…
There are numerous ways to find treatments for rare diseases, with the major method that springs into everyone's minds being clinical trials starting from scratch. However, in many cases, drugs…
Understanding rare diseases is integral in treating them. In order to do so, research into the underlying causes and factors behind these conditions is needed. A recent study published in…
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