Cannabidiol (CBD) has recently been lauded for its many uses, ranging from stress relief to seizure reductions. Specialty pharmaceuticals company Zynerba Pharmaceuticals (“Zynerba”) developed an experimental cannabidiol gel called Zygel…
Early detection is the key to treating so many types of cancer. Autoimmune hepatitis (AIH), chronic inflammation of the liver, is brought about by an immune system disorder. AIH must…
What do medication, vagus nerve stimulation, and low-carb ketogenic diets all have in common? Currently, these are all therapeutic options for those with Dravet syndrome. However, researchers and doctors believe…
In a news release from January 24, 2022, immuno-pharma company AIM ImmunoTech Inc. ("AIM") shared that positive clinical data was now published. The data, which you can find in Clinical Cancer…
Oscar Warrent, a toddler from Norwich, England, has been through a lot in his two years of life. About three months ago, he was diagnosed with rhabdomyosarcoma, a rare form…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…
According to a recent article, new evidence shows adding ibrutinib to a patient’s chemotherapy regimen may improve the longevity of younger patients with diffuse large B-cell lymphoma (DLBCL). Diffuse Large…
Glioblastoma is a highly malignant and aggressive brain cancer. With a 5-year survival rate of under 10%, there is an urgent need for newer and more effective diagnostic and treatment…
According to a recent article, the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition displayed the advances that have been made in the management of follicular lymphoma. Follicular…
We have been living in the midst of a pandemic for years now, and with that has come a number of lasting effects. One of these effects, which has been…
According to a recent article, patients diagnosed with minimal change disease are found to have autoantibodies targeting nephrin, which supports previous research. Minimal Change Disease (MCD) Minimal change disease is…
Over the last few years, more research has been dedicated to understanding the impact of gut health and the gut microbiome on overall health. More recently, researchers sought to understand…
Understanding a rare disease is the first step in treating it. How can you combat the underlying mechanisms of a disorder if you do not know what they are? A…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Cannabidiol (CBD) is a chemical compound found in cannabis and derived from hemp. Unlike THC, CBD is not psychoactive. It is lauded for its ability to treat a wide variety…
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a rare group of hereditary neurodegenerative…
According to a news release from immuno-oncology company IMV Inc., the first patient has been dosed in the Phase 2b VITALIZE clinical trial. During the trial, researchers are evaluating a…
Over the last two years, the COVID-19 pandemic has dominated the headlines. In 2021, vaccines became available to prevent severe COVID-19 infection. However, this has also brought up numerous questions…
Moebius Syndrome Awareness Day is January 24th! This event was created in 2011 by the Many Faces of Moebius Syndrome (MFOMS). It is now celebrated across the world, as the…
In late December 2021, biopharmaceutical company Deciphera Pharmaceuticals, Inc. ("Deciphera") shared that its therapy QINLOCK (ripretinib) was approved in the United Kingdom. The treatment is designed for adults with advanced…
BeyondSpring Pharmaceuticals has issued a press release announcing that the FDA presented the company with a complete response letter pertaining to its drug plinabulin and granulocyte colony-stimulating factor (G-CSF). The…
The Klassen family is no stranger to the rare disease community. According to the Denver Post, Greg Klassen was first diagnosed with spinocerebellar ataxia type 3 (SCA3) nearly ten years…
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
According to a recent article, Castle Creek Inc. has acquired Novavita Thera Inc. in an effort to expand its research and development efforts. Castle Creek Inc. Castle Creek Biosciences Inc.…
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