According to a story from BioSpace, the biotechnology company Cellenkos, Inc., recently announced that the US Food and Drug Administration (FDA) has given the company the go ahead to begin…
The 2018 FH Global Summit featured fantastic speakers, timely information, and valuable insight about the past, present and future of the way FH is understood, diagnosed, and managed. The summit…
According to a story from markets.businessinsider.com, the drug development company Pharming Group N.V. recently announced results from a Phase II trial testing its medication RUCONEST. This drug was tested as…
Genomic testing is becoming increasingly common, but more work still needs to be done into understanding which gene variants are linked to health and disease. To address this, the National Institutes…
The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease…
Across the globe, countries are transitioning their healthcare systems to provide more care within the patient's home and less in a hospital setting. Why? Simply put, it's because there's beginning…
British Columbia faces excruciating decisions about how and whether to cover drug treatment for rare diseases. One particular drug is Spinraza. It is a drug that is a part of…
Happy Friday everyone! The leaves are beginning to change, and so are ongoing activities at the FDA. This week, we're highlighting four FDA announcements we think you should hear about.…
This story contains images of severe lesions and blistering. On the weekend of October 12-14th of 2018, Patient Worthy had the privilege of attending the International Pemphigus & Pemphigoid Foundation's …
According to a story from Market Screener, the pharmaceutical company Retrotope has recently released data that indicates that the company's experimental product RT001 is capable of halting disease progression for…
According to a story from Charcot-Marie-Tooth News, a recent meeting between the US Food and Drug Administration (FDA) and patient advocacy groups took place to discuss the future development of…
According to a story from the New York Times, Harvard University has recently called for the retraction of 31 studies authored by a cardiologist associated with the school. The cardiologist…
Don't miss this special two day event made possible by The Myelodysplastic Syndromes Foundation , Sylvester Comprehensive Cancer Center at the University of Miami and The Aplastic Anemia and MDS International Foundation MDS…
The Undiagnosed Diseases Network (UDN) is working to help patients with rare undiagnosed conditions find answers. So far, they have helped more than two hundred patients achieve a diagnosis. About…
October is Achondroplasia (Dwarfism) Awareness Month! Achondroplasia is a bone growth disorder that causes dwarfism. Dwarfism is defined as a condition of short stature as an adult. People with achondroplasia are…
Increasingly, researchers are looking into how the human microbiome – the trillions of microbes that live on and in your body – can influence various aspects of health, from nutrition…
The Patient Advocate Foundation has recently begun to expand the reach of its popular Co-Pay Relief program. The Co-Pay Relief program is designed to provide financial assistance to qualifying patients.…
According to a story from pm360online.com, the orphan lung disease company Savara recently announced that they had completed their enrollment target of 135 patients for a Phase 3 clinical trial…
According to a story from Roche, the pharmaceutical company recently released new data from its ongoing clinical trial, which is testing the company's investigational product risdiplam. In this study, the…
According to a story from the Washington Post, new cases of the unusual, polio-like disease called acute flaccid myelitis have continued to appear through the United States recently. There are…
The Parkinson’s Institute and Clinical Center and Retrotope are teaming up to study a potential new therapy for patients with progressive supranuclear palsy. For more information about this collaboration, you…
"Five to 10 percent of all cancer is hereditary" - Adelsperger Genetic counselors can help you understand not only your own risk for developing cancer but the risk of your…
According to a story from BioSpace, the biotechnology company X4 Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's investigational…
According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…
US Congresswoman Lucille Roybal-Allard gave a recent speech in November raising awareness about a looming health crisis for US people. Nonalcoholic steatohepatitis (or NASH), she declares, is looming in the…
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