For patients with refractory or relapsed KMT2A rearrangement or NPM1-mutations, the AUGMENT 101 study of the investigational menin inhibitor revumenib yielded impressive results. According to an article in MedicalXpress,…
Clinical trials are often mentioned as an afterthought when discussing the length of time it takes for drugs to be approved by the FDA. On the contrary, clinical trials are…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
Throughout her life, Hallie Hale has always been drawn to dance. There was something equally challenging and relaxing, hypnotic and focused, about what she could achieve. Her parents knew…
3-year-old Ava Bolton has always been bright, vivacious, and gregarious. So her parents Natalie and Scott were understandably worried when, near her third birthday, Ava's affect seemed to change. According…
In a late April 2023 news release, biotechnology company UNITY Biotechnology, Inc. ("UNITY") shared that the company had observed positive long-term follow-up data from the Phase 2 BEHOLD trial. Within…
According to an article published in Cold Agglutinin News, Dianthus Therapeutics (“Dianthus”) is planning on holding a proof-of-concept study in 2024. Within the study, the company would evaluate the safety…
In December 2022, Representative Jamie Raskin (D-MD) shared with the public that he had been diagnosed with diffuse large B-cell lymphoma (DLBCL). Although being diagnosed with cancer can be frightening,…
People with severe cases of Eastern equine encephalitis (EEE) or Venezuelan equine encephalitis (VEE) can develop significant brain inflammation, causing damage. An estimated 33% of people with severe EEE infection,…
This weekend during the Leiden Marathon, more than 30 parents, children, doctors, researchers and members of the public will run to raise money for the Well Bee Study. This important…
In the past, budesonide has been used in a number of therapeutic indications, such as to reduce proteinuria in IgA nephropathy or digestive tract inflammation in those with Crohn's disease.…
Have you been struggling with gastrointestinal (GI) issues? Does your stomach ache? Have you been throwing up or experiencing diarrhea—but you’re not quite sure why? Well, you might have alpha-gal…
In 2021, Josie Gosney entered the world and her parents couldn’t be more thrilled. When she was first born, Josie showed signs that something might be right. But after a…
It all began at a movie theater. Kurt Harrington and his father were leaving the theater when his father decided to hold the door open for some others. Someone knocked…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…
In the past, doctors and researchers surmised that having chronic kidney disease (CKD) could increase the risk of having a stroke. Yet they didn’t know exactly why it happened or…
It can be tough to deal with a cancer diagnosis. After all, the diagnosis comes with a huge emotional and psychological impact, not to mention the uncertainty about the future…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The citizens of Cincinnati know one universal truth: that the annual Flying Pig Marathon weekend will bring fun, family, and fulfillment to their city. And with that universal truth comes…
Usher syndrome is a rare genetic disorder that is characterized by deafness and often paired with retinitis pigmentosa (RP), an inherited disease which causes retinal degeneration and vision loss. There…
During the REPREVIO clinical study, researchers sought to understand whether using vedolizumab (Entyvio) after bowel resection could prevent the risk of recurrence in individuals with advanced Crohn’s disease. Participants were…
Written by Carter Hemion On April 17, 2023, Governor Inslee signed a ceremonial proclamation recognizing May as Ehlers-Danlos Syndromes and Hypermobility Spectrum Disorder Awareness month in Washington State. The International…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
There are still many unknowns when it comes to our genetics. How do gene variants of unknown significance (VUS) affect us? In what ways can we identify if variants…
The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
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