A new partnership has been formed between Global Genes and the Rare Disease Diversity Coalition (RDDC) - created by the Black Women's Health Imperative - in an effort to increase…
A lack of awareness is a problem that many rare diseases face. Small patient populations, limited research, and other factors all play a role in this issue. Luckily, there are…
Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
The 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) was held last week from the 13th to the 15th. This event is the largest…
Now that it's officially October, it's time to celebrate my personal favorite holiday - Halloween. That means haunted houses, scary movies, creative costumes, and spooky decorations. One Transcona family is…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
Rare disease therapies are often difficult to access, whether that is due to price, a patient's location, or another factor. Because of these obstacles, many rare disease patients find themselves…
Last week marked National Trigeminal Neuralgia Awareness Day, which was celebrated on October 7th. It's meant to raise awareness of this rare disease while providing support to those affected. You…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
The first patient in a Phase 2a trial of BOS-580 as a treatment for nonalcoholic steatohepatitis (NASH) was recently dosed. Boston Pharmaceuticals, the developer of the drug, aims to address…
Explaining something as complicated as a rare disease to a child can be difficult. That's why Alexion Pharmaceuticals created a books series titled "Inspired By." The series is a way…
A rare disease diagnosis can change your life; Mari Jackson knows this firsthand. She received a surprise diagnosis of pulmonary arterial hypertension (PAH) at age 48. Her symptoms began suddenly,…
According to a recent press release from Horizon Therapeutics, UPLINZA, the only FDA-approved treatment for AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), may be a viable treatment option for African…
Clinical trials face many obstacles regardless of which disease they aim to investigate. However, rare disease trials face additional problems that don't typically impact studies for more common conditions. Small…
"Rare" is a documentary that gives its viewers a glimpse into the world of rare diseases, something that many people know little about. Until now, the project has been funded…
Losing a loved one can be life-shattering. Ginny Toman knows this, as she lost her father to progressive supranuclear palsy (PSP) in 2019. Since then, she has learned how to…
A new film is set to premiere tomorrow, and it depicts the life of Jeff Golub. This famous musician was known for his talent on the guitar, but what many…
Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…
A recent study conducted by the Penn State College of Medicine revealed that taking glucagon-like peptide-1 receptor (GLP-1R) agonists six months before COVID-19 infection decreases the risk of a severe…
A new collaboration between the University of Texas's MD Anderson Cancer Center and the Rare Cancer Research Foundation aims to solve a problem that has been a major obstacle for…
Long QT syndrome (LQTS) is a rare condition that's characterized by fast and chaotic heartbeats. Episodes have the potential to cause fainting, seizures, or even death. Because of this, affected…
It's a beautiful thing when a community pulls together to support one of its members. This is exactly what the Madeira community is doing for 16-year-old Diego Ramirez. He recently…
Lytle, Texas is a small town located outside of San Antonio. Recently, the entire community came together to support a young boy living with autosomal recessive polycystic kidney disease (ARPKD).…
Alan Jackson has been a big part of the country music scene for a long time, with songs like Remember When and Livin' On Love. Recently, he revealed to his…
Having a rare disease can feel isolating. Many people have never even heard of your condition, much less know anything about it. This is even a problem with doctors. Despite…
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