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…
A recent discussion between Dr Crystal Mackall and Dr Lynn Zydowsky explored the research, challenges, and future of cancer gene therapies. Dr Mackall is a doctor and renowned cancer researcher…
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…
If you have a health condition where you are misunderstood and perhaps judged by others for how you live your life to battle your symptoms, I invite you to read…
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…
uBiome is holding a challenge that encourages people to submit proposals for a chance to win a grant. The challenge, called the Clinical Outcomes in Microbial Genomics Innovation Challenge, is…
October is Kabuki Syndrome Awareness Month! And it's important to connect with other people in the community; there's always strength in numbers. So let's learn a little more about Kabuki syndrome…
What is PML? PML stands for progressive multifocal leukoencephalopathy. It’s a brain infection caused by the JC virus. The JC virus itself isn’t all that rare. In fact, its pretty…
According to a story from Bioportfolio, the biopharmaceutical company Newron Pharmaceuticals has chosen to support Rett Syndrome Awareness Month this year. Rett Syndrome Awareness Months falls on the October of…
Zealand Pharma A/S (Zealand) recently announced in a press release the start phase 3 trial for short bowel syndrome treatment. The trial investigates the use of glepaglutide as a treatment…
According to a story from OncLive, the drug development company Karyopharm Therapeutics recently announced that the US Food and Drug Administration has granted Priority Review to the company's XPO1 inhibitor…
According to a story from news-medical.net, Verastem, Inc., recently announced that its drug COPIKTRA (duvelisib) was recently added to the National Comprehensive Cancer Network's (NCCN) guidelines for the treatment of…
As part of their work to support the treatment of patients with multiple sclerosis, Biogen has been investigating a potential new biomarker for the disease called serum neurofilament light (sNfL),…
The United States Food and Drug Administration has accepted an Investigational New Drug application (IND) for an investigational gene therapy designed to be a treatment for sickle cell disease. Previously,…
According to a report by BioSpace, the United Kingdom’s National Institute’s of Health (NIH) recently diagnosed 31 conditions that were not previously recognized. The results date back to an initiative…
A clinical trial application for a Phase 1 study of the investigational drug KL1333 in healthy volunteers and patients with genetic mitochondrial diseases has been approved by a regulatory agency…
Death or Debt? That is the question that begins the title of a recent study that is examining "financial toxicity" for patients with newly diagnosed cancer. Treatment for cancer is…
According to a story from PMLive, Leadiant Biosciences of Italy recently announced that its drug Revcovi has officially received approval from the US Food and Drug Administration. Revcovi was approved…
According to a story from Endpoints News, the Chief Science Officer for Spark Therapeutics is close to finishing up his first year with the company. His name is Federico Mingozzi,…
According to a story from Charcot-Marie-Tooth News, the continuously escalating cost of drugs in the United States is still plaguing rare disease patients and chronically ill people across the country.…
Some of you may have heard this story, others may be completely new to this, but bear with us on this funny and on-topic story that made the rounds on…
When it comes to rare diseases often one of the biggest hurdles is finding the right diagnosis. Tailoring treatment, and adjusting lifestyles can be difficult, but both are impossible if…
According to a story from mirror.co.uk, Lee and Charmaine Taggart of Kent are the parents of Martha, a six year old girl who was born with Angelman syndrome. Martha's diagnosis…
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