A recent study has documented the differences between two rare forms of childhood brain inflammation. The two similarly presenting conditions are often confused for one another. A better understanding of…
Mike Jackson's young daughter Evelyn was diagnosed with Batten disease after a neurologist suggested a genetic test which the family could take for free. John Barclay's daughter River passed away…
In 2015, the FDA approved Apollo Endosurgery, Inc.'s Orbera Intragastric Balloon for weight loss. Now, the company shares in a press release, the device recently received Breakthrough Device Designation…
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…
For more than 30 years, Ionis Pharmaceuticals, Inc. ("Ionis") has worked to create RNA-targeted therapies for patients in need. Now, according to a recent press release, the company initiated…
Some cancers and hematological conditions can be difficult to treat. But biotechnology company Oncopeptides AB is working to address this issue for patients with relapsed or refractory (R/R) multiple…
The FDA's Orphan Drug designation offers a special status to drugs or biologics designed to treat rare conditions. In the United States, a rare disease is one that impacts less…
According to BioSpace, Kronos Bio will continue with its plans for a Phase 3 trial of entospletinib after a positive meeting with the FDA. One of the meeting's focuses was…
A recent study published in Cell Reports has uncovered a mechanism where germs living within the lungs of cystic fibrosis (CF) patients produce a type of carbohydrate slime that minimizes the efficacy…
In a recent press release, biopharmaceutical company Salarius Pharmaceuticals, Inc. ("Salarius") shared that it had initiated an expansion stage of a Phase 1/2 clinical trial. Within the trial and the…
James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to…
A new study published in JAMA Oncology has found that of all patients who have had cancer spread to their bones and started treatment, 2,8% have developed osteonecrosis within their jaw, also…
For patients with severe atopic dermatitis, it can be difficult to find adequate treatment options. For many, treatment revolves around topical creams, anti-inflammatory medications, light therapy, antibiotics, and more. But…
The ongoing COVID-19 pandemic has thrown the world into chaos. With over 500,000 dead in the US alone, the impacts of this global health crisis have affected many different facets…
Recently, biopharmaceutical company Strongbridge Biopharma plc ("Strongbridge") shared that the company submitted a New Drug Application (NDA) to the FDA. The NDA is centered around RECORLEV (levoketoconazole), a treatment option…
Researchers from Northwestern University have discovered a compound with the ability to stop the progressive degeneration of motor neurons in those with amyotrophic lateral sclerosis (ALS). The continued breakdown of…
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…
In a press release from March 3, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared that it had completed full enrollment for a Phase 2 clinical trial evaluating vosoritide for…
During the National Institutes of Health (NIH) Rare Disease Day at NIH event, a panel was held titled "From Kitchen Tables to Changing Paradigms — Advocacy as a Driving Force…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
In a joint statement, biopharmaceutical companies AstraZeneca and Merck & Co., Inc. ("Merck") shared that LYNPARZA (olaparib) achieved a superiority boundary in the Phase 3 OlympiA clinical trial. During…
Supporting the development of my children's talents has returned the joy of life to our whole family - in spite of HAE diagnosis and other obstacles in life. As you…
Like many other residents of West Hollywood, Mark Chaney uses public transportation, specifically the bus. Twice a day, Monday through Friday, Mark boards the bus at his local stop. He…
As reported in NewsWise, as rare diseases get their time under the microscope, growing evidence uncovers medical distinctions. New lines are drawn, delineating new disorders to be defined and classified.…
Monty Hui, an eight-year-old boy from Australia, and his family have spoken to SBS News about living with a rare disease and the obstacles faced in diagnosis and recognition. He…
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