According to a story from BioPortfolio, the biopharmaceutical company Deciphera Pharmaceuticals, Inc., recently announced the release of preliminary data from the company's Phase I clinical trial, which is still in…
Multimorbidity, typically defined as having two or more long-term health conditions, has become a topic of increasing focus for healthcare providers. Many elderly people are affected by multimorbidity, with the…
According to a story from pm360online.com, the biopharmaceutical company Ovid Therapeutics recently announced that it had received the 2018 Company Making a Difference Award from the CDKL5 Forum.This recognition was given to…
An ambitious project called the Human Cell Atlas is attempting to study all the cells in the human body – of which there are more than 37 trillion. Researchers hope…
According to a story from crainsnewyork.com, the biotechnology company Gotham Therapeutics has received financing to the tune of $54 million so that it can begin developing drugs to treat rare…
I remember being 16 years old and commencing my first drug to help manage my heart rate and endless dizziness. It was strange, because I recall asking my friends if…
According to a story from BioSpace, EUSA Pharma and AVEO Oncology recently announced the release of updated interim results from the second Phase of a Phase Ib/2 clinical trial which…
If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of…
According to a story from Science Daily, a group of scientists from the Australian National University have managed to discovery a new genetically linked disease and have also pioneered a…
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…
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