In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
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…
Earlier this year, WP1066, a p-STAT3 inhibitor, received three separate Rare Pediatric Disease designations for the treatment of medulloblastoma, atypical teratoid rhabdoid tumor, and diffuse intrinsic pontine glioma. Now,…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
Whitney Dafoe was an aspiring photographer who backpacked across the world. However, he always would return from his trips extremely exhausted. He would feel weak and he didn't know why.…
According to Drug Target Reviews, there may be an emerging vaccination effective against preventing Zika virus infection. Though many cases are mild, the infection can cause severe birth defects and…
A recent study published in Frontiers in Immunology investigates the immune response to bone infections, such as Staphylococcus aureus, that cause osteomyelitis. Their research stands to give medical professionals a better…
On April 1, 2021, biopharmaceutical company AbbVie shared that new data is available from the Phase 3 SELECT-PsA 1 clinical trial. The 24-week results follow the impact of RINVOQ (upadacitinib)…
In late March 2021, the FDA gave Fast Track designation to annamycin. The treatment, initially developed for patients with acute myeloid leukemia (AML), is now being explored for patients with…
#KnowThyNuts. While the hashtag, on its surface, sounds a bit funny, it is actually a call to action. According to a news release from Movember Canada, testicular cancer, though…
Severe Combined Immunodeficiency Severe combined immunodeficiency (SCID) is a group of diseases caused by mutations that affect how the immune system works to fight infections. These anomalies are usually passed…
Could repurposing certain drugs help treat other conditions? Well, according to Technology Networks, yes. Recently, researchers from the UCL Great Ormond Institute of Child Health and the Great Ormond Street…
Recently the Leukemia & Lymphoma Society (LLS), in coordination with the International Waldenstrom's Macroglobulinemia Foundation (IWMF), hosted its Waldenström's Macrogloblinemia (WM) Patient Education Program webinar on April 7th, 2021. Patient…
What if we could harness a naturally occurring compound to starve and kill serious brain tumors? According to Medical XPress, researchers from Queen Mary University of London are working to…
A study published in Physician's Weekly investigated the visual outcomes, clinical features, and treatment of individuals with occlusive retinal vasculitis (ORV), a condition that is characterized by inflammation in the vessels of…
A variety of gene mutations cause retinitis pigmentosa (RP) - but how do those genes affect the degeneration of rod and cone photoreceptors in the eyes? According to Medical XPress,…
According to a story from Fabry Disease News, the month of April is recognized as Fabry Disease Awareness Month, a time dedicated to spreading awareness about Fabry disease in the…
In a recent press release, immuno-oncology and biopharmaceutical company GT Biopharma, Inc. ("GT") shared the release of new data and results from the Phase 1/2 GTB-3550 TriKE clinical trial. Through…
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…
I think it would be safe to say that everyone is afraid of something. Sometimes the fear is justified and sometimes not. In this article, I want to focus on…
According to a story from Pompe Disease News, April 15th will be recognized as the eighth annual International Pompe Day. This day is meant to spread awareness about Pompe disease…
As many patients within the rare disease community know, the diagnostic odyssey can be long and arduous. But what if burgeoning technologies, like artificial intelligence (AI) and its branches (machine…
One of six-year-old Jackson Garwood's favorite things to do is listen to his father sing. Some of his favorite numbers include 'Talk Tonight' by Oasis and Aqualung's 'Brighter Than The…
From April 5 - 30, the American Chemical Society meets to hold ACS Spring 2021, where chemistry professionals meet to discuss new research and knowledge in their field. During the…
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