In a video conversation recorded for Cancer Network, Kristie L. Kahl and Javier L. Munoz, MD, MS, FACP discussed treatment for relapsing/remitting follicular lymphoma and the importance of the sequencing…
Have you ever heard of Bachmann-Bupp Syndrome (BABS)? If not, don't worry, you're not alone. In fact, BABS was only first discovered over the past few years. According to Medical…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The very first HAE symptom I had brought me to the operating table. Unnecessarily. It was summer, with holidays and grandmother's birthday party- a big family reunion in the garden.…
Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen,…
If you could be vaccinated against serious conditions via nose drops, would you take that option? Well, it could one day be a possibility. According to New Atlas, scientists from…
According to a story from USA Today, Mark Hoppus, bass player for the iconic pop-punk band Blink-182, was recently diagnosed with a rare cancer: diffuse large B-cell lymphoma. His cancer…
According to a news release from late June 2021, biopharmaceutical company Pharming Group N.V. ("Pharming") completed enrollment for a Phase 2/3 clinical trial evaluating leniolisib for patients with activated phosphoinositide…
OncLive recently interviewed Daniel Pollyea, MD, MS, about the current treatment landscape for higher-risk myelodysplastic syndromes (HR-MDS), along with exciting new data from a Phase 2 trial of pevonedistat plus…
Researchers from Boston University and Boston Medical Center have recently uncovered that base editing is able to correct the gene mutation which causes alpha-1 antitrypsin deficiency (A1AD). This condition impacts…
While myasthenia gravis (MG), an autoimmune neuromuscular disorder, can be treated, there is a smaller subset of patients who are treatment-refractory. Those whose condition is not easily treated are thought…
The name of the organization is Disease InfoSearch. It is not only a resource for credible information but guides patients and families to valuable support groups and participation in…
In the company's Phase 2 proof-of-concept clinical trial, biopharmaceutical company Harbour BioMed ("Harbour") sought to understand the impact of batoclimab (HBM9161) on patients with generalized myasthenia gravis (gMG). The…
If you have ever donated to the American Red Cross, you've probably been receiving a rash of calls and emails asking for donations. I know I have. This is because…
In the past, Crysvita was approved for subcutaneous administration by a physician for patients with X-linked hypophosphatemia (XLH). This injection could be both invasive and time-consuming. However, according to the…
This question is what the doctors at Massachusetts’ Cambridge Hospital were trying to determine. According to a recent article in the New York Times, a twenty-two-year-old man was rushed to…
Community support can be extremely uplifting and important, especially when one is impacted by a rare disease. This is the case for three-year-old Charlie Fry, who was born with SLC6a1.…
A study conducted by researchers at Nagoya University in Japan and researchers from Yale University in the United States was recently published in the European Respiratory Journal. This study utilized single-cell…
Clinical trials can be crucial in determining potential treatment options for a variety of conditions. According to Pharma Biz, biopharmaceutical company Nordic Nanovector ("Nordic") recently shared promising data from the…
It's the season of sun and summer fun, which leads many to enjoy the great outdoors and warm weather. While this is all in good fun, it's important to remember…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The patient sitting in front of this physician was later diagnosed with severe chronic fatigue syndrome (myalgic encephalomyelitis or ME/CFS). But for now, the patient had to endure humiliation…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
In a study published in the research journal Scientific Reports, CD71+ erythroid cells (CECs) have an immunoregulatory function. They often appear in people with cardiopulmonary or hemolytic disorders, as well as in…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
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