Following Rare Disease Day at the NIH Clinical Center in early March, we spoke to the NIH about remaining questions we had pertaining to rare disease research. We are impressed with…
In the beginning of March, Patient Worthy joined many other groups from the rare disease community for Rare Disease Day at the NIH Clinical Center, an event which you can…
According to a story from news-medical.net, the Genomics England company, which was founded as a subsidiary of the UK's Department of Health and Social Care in 2013, announced recently that…
Sisters Nicole and Jessica Rich, both diagnosed with Batten disease, will finally be able to receive treatment at Great Ormond Street Hospital in London. Nicole, who is six years old,…
According to a story from the International Business Times, a total of eleven new medications are about to become a whole lot cheaper for Australia's citizens. Slated to be added…
According to a story from Business Wire, there was a time when having to use a mechanical ventilator in order to breathe meant being confined at the hospital in an…
According to a story from cbc.ca, Lilia Zaharieva, a woman from British Columbia with cystic fibrosis, was recently given long term access to a treatment that is currently saving her…
According to a story from cnbc.com, the pharmaceutical company Novartis recently announced that their anticancer drug Tasigna has been approved for the treatment of patients one year or older that…
According to a story from Financial Content, the drug company Arena Pharmaceuticals recently announced that its data results from Phase II clinical trials has been positive. The trial in question…
According to a story from CBS DFW, doctors have begun to test the use of fluorescent dyes in order to detect hidden cancer tumors. So far, the dyes appear to…
March is not just multiple myeloma awareness month... ...it is multiple myeloma ACTION month! Multiple myeloma is a form of cancer that develops in a type of white blood cell…
According to a story from cbc.ca, a recent study suggests that the benefits of summertime sun exposure may be more effective at preventing multiple sclerosis symptoms than previously understood. Prior…
According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…
According to a story from globenewswire.com, the pharmaceutical company Amicus Therapeutics recently announced that its drug Galafold had received official approval for public use in Japan. Galafold is the first…
According to a story from pressreleasepoint.com, a group of scientists at the University of Birmingham have a received funding to the tune of £3 million in order to conduct research on…
According to a story from Digital Trends, Google has recently added an option for wheelchair-accessible routes on its widely popular navigation tool, Google Maps. This option will make the app…
You wouldn't know by looking at 5-year-old Grayson Ledbetter that he only had a few years of life left to live. According to The Washington Post, Grayson battles the rare,…
Erin Gard and her mother, Joanne Guthrie-Gard, already spent many years of their lives searching for an effective epilepsy medication for Erin. However, once they found it, they lost access…
According to a story from medscape.com, the 2018 budget recently signed by the president is going to feature some spending increases for health-related services. These changes have the potential to…
According to a story from the National Institutes of Health, the innovative new CRISPR gene editing technology is being used in order to identify specific genes that could play a…
According to a story from biospace.com, a research team from the National Cancer Institute was able to successfully deliver an experimental nanomedicine to mice that had rare cancers such as…
According to a story from Medical Xpress, two new treatments were able to slow down the worsening of symptoms associated with muscular dystrophy in a mouse model study. Tamoxifen and…
According to the New York Post, 16-year-old Brandon Marshall from the UK stands tall at a whopping 7' 4", possibly making him the tallest teen currently in the world. His…
Anna Bridgeman, mother from Redbank Plains, luckily avoided death despite experiencing incredibly low kidney function, reported The Queensland Times. Anna had gone through much of her life without any serious…
Vanderbilt University Medical Center researchers have discovered a new method of searching existing electronic medical records to help identify genetic diseases in larger populations, reported EurekAlert!. Many people who may…
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