.png)
Since the 1940s oncologists have been focusing on tumors at the location in the body where the tumors originated. According to a recent FDA press release, this is the third…
According to a story from forextv.com, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND)…
According to a publication from CURE Today, the US Food and Drug Administration (FDA) recently approved Genentech, Inc's Rozlytrek (generic name entrectinib) for the treatment of cancer patients who have…
According to a story from uk.finance.yahoo.com, the company Hutchison China MediTech Limited ("Chi-Med") has recently kicked off a phase 1 clinical trial. This trial is testing the company's investigational drug…
According to a story from Markets Insider, Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has approved the company's drug ixekizumab (marketed as Taltz®)…
Netflix debuted a documentary series this month called 'Diagnosis' -- based on a New York Times Magazine column -- which follows individuals with or caregiving for patients with a mysterious,…
According to a story from Biotech 365, the biotechnology company Stoke Therapeutics, Inc., recently announced that the very first patient has enrolled for the company's observational study of Dravet syndrome…
According to a story from Healio, a recent study has illustrated that the drug denosumab can improve the bone density of patients with primary biliary cholangitis (PBC) and autoimmune hepatitis…
According to a press release from Synlogic Therapeutics, the Company recently halted development of its investigational hyperammonemia treatment SYNB1020 after an unsuccessful phase 1/2 clinical study. About Hyperammonemia Hyperammonemia is…
Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…
According to a story from abc.net.au, an unusual and poorly known rare disease has been making its presence known in the country of Australia. This disease is called mal de…
According to a story from Financial Buzz, the Steadman Philippon Research Institute (SPRI) recently received an award in research from the National Institutes of Health (NIH). The award is in…
According to a story from Mizzou News, Chris Lorson, who is the associate dean for Research and Graduate Studies at the school's College of Veterinary Medicine, recently earned four grants…
According to a story from Cancer Research UK, a combination treatment featuring the targeted cancer therapy venetoclax (marketed as Venclyxto) and the monoclonal antibody rituximab will soon be covered by…
TGIF! Something we believe is that the stories of rare disease patients are vitally important. This week, we're highlighting two articles written by patients, as well as a story about…
According to a story from Market Watch, the biotechnology company Vertex Pharmaceuticals, Inc. has announced recently that the US Food and Drug Administration (FDA) has accepted the company's New Drug…
According to a story from PR Newswire, the biopharmaceutical company Harmony Biosciences, LLC recently announced that the US Food and Drug Administration (FDA) has approved the company's drug pitolisant (marketed…
Sumaira Foundation Sumaira Ahmed established the Sumaira Foundation so that fewer rare disease patients would have to face the experience by themselves. Sumaira Ahmed is diagnosed with neuromyelitis optica spectrum disorder…
Here's a shocking fact for you- individuals don't always conform to averages. Despite this, we use averages as an analysis for so many things. As it documents the majority, its…
According to a story from Angioedema News, the drug development company Regenxbio has announced that it is in the process of developing a new gene therapy treatment for hereditary angioedema,…
According to a story from BioPortfolio, the biopharmaceutical company Emalex Biosciences, Inc., has recently announced that the very first patient has been signed up for the company's phase 2b clinical…
According to a story from The People's Pharmacy, the health dedicated website (along with many other sites that discuss drug effects and side effects) has experienced a nearly 50 percent…
According to a publication from BioSpace, the American Food and Drug Administration (FDA) recently approved the Inrebic (fedratinib) for the treatment of certain myelofibrosis patients. About Myelofibrosis Myelofibrosis is a…
According to a recent article in CheckOrphan, glioblastoma (GBM) is considered to be among the deadliest cancers in the world. Currently, treatment options are surgery, chemotherapy, and radiation. In most cases,…
According to a story from Pharma Times, the drug company Ipsen has recently made available a pre-filled syringe of its drug lanreotide (marketed as Somatuline) which will allow patients to…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
