Mitochondrial Disease Mitochondrial disease is a rare and progressive condition that currently does not have a cure. The mitochondria is responsible for providing energy to the body. This energy is used…
There are thousands of rare diseases, making it difficult to remember, much less understand them all. Patient Worthy is here to help, explaining and raising awareness for rare conditions. Today…
Dr. Andrew Brunner, an oncologist at Massachusetts Cancer Center was recently interviewed by Targeted Oncology. The discussion centered around a phase 1b clinical trial (NCT03066648) investigating sabatolimab together with hypomethylating…
Soleno Therapeutics has announced that they will be hosting a Key Opinion Leader (KOL) webinar on Thursday, February 4 at noon EST. This webinar will focus on Prader-Willi syndrome, specifically…
The UK has recently began the practice of the new UK Rare Disease Framework, a government effort that aims to spread awareness, quicken the diagnostic process, and improve treatment and…
A new licensing and supply deal has been made between Mitsubishi Tanabe Pharma America Inc. (MTPA) and Aquestive Therapeutics concerning the commercialization of riluzole in the United States. Riluzole, being…
Parkinson’s News Today recently featured an upbeat article describing the progress that researchers have made in developing a method of measuring alpha-synuclein’s (AS) progression that cuts discovery time in…
Kuur Therapeutics has recently released interim data on their CAR-NKT cell therapy platform, which was created for the treatment of hematological and solid malignancies. This data comes from two phase…
According to a story from Business Weekly, the biotechnology company Healx, from Cambridge, UK, and the US-based Mission: Cure coalition have announced a new partnership which aims to help develop…
According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat.…
The National Institute for Health and Clinical Excellence (NICE), an agency under the UK's National Health Service (NHS), has recently rejected Zeposia. Zeposia is an oral, multiple sclerosis (MS) treatment…
Although advances in lung cancer care were overshadowed this year by news about the pandemic, Dr. Jennifer Carlisle’s recent interview with Oncology gave details of spectacular advances in lung cancer…
According to a story from Yahoo! Finance, EUSA Pharma Limited and BeiGene, Ltd. have recently announced that their Biologics License Application (BLA) has been accepted by the China National Medical…
Sorafenib, a tyrosine kinase inhibitor, has been unbeatable for treating cancer of the liver for the past thirteen years. An article by the Cleveland Clinic stated that during that time,…
According to an article published in Medscape, guidelines for the treatment of valvular heart disease have changed. The American College of Cardiology (ACC), along with the American Heart Association (AHA),…
Over the past decade, scientists have made many life-saving advances in all areas of medicine. They have created molecular agents to target neurodegenerative diseases. But they were stopped right there…
Oftentimes with cancers or rare conditions, there are subtypes of the cancer or disease. Breast cancer is no different. For example, a breast cancer diagnosis may encompass BRCA gene mutations or triple-negative…
According to a story from newswise.com, a team of researchers from the University of New Hampshire may have discovered the next new treatment for a rare form of blood cancer…
Patient input is extremely important when evaluating treatments for rare diseases; Charcot-Marie-Tooth disease (CMT) is no exception. In order to understand the impact of different CMT therapies, researchers from the…
The medical community has been aware for some time now that patient involvement in his or her treatment is a critical part of therapeutic development. Biopharmaceutical companies acknowledge their obligation…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
Written by Kendra J. Bjoraker, Ph.D., L.P. Living well with a chronic condition such as Fabry can seem contradictory. Following diagnosis, you don’t have a shelf in your mind that…
In late December 2020, biopharmaceutical company AbbVie announced via press release that the prescribing information for IMBRUVICA (ibrutinib) is now updated to include long-term safety and efficacy data for…
On January 10, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared positive data from its Phase 3 GENEr8-1 clinical trial. The clinical trial is evaluating an investigational gene therapy…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
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