According to a story from finanznachrichten.de, the biopharmaceutical company Abeona Therapeutics, Inc. recently announced that its experimental medical product ABO-101 has earned Fast Track designation from the US Food and…
According to the publication from the London Free Press, a new technique in development at Lawson Health Research Institute uses epigenetics to more closely study genetic expression in humans. Epigenetics…
The Burdens of Chronic Disease There are many types of chronic diseases, affecting patients in all kinds of ways. However chronic conditions share one thing in common- they all pose…
Idiopathic pulmonary fibrosis (IPF) is a rare condition with no known cause and no effective treatments besides a lung transplant. Current therapies focus on slowing progression of the disease but…
.jpeg)
You will never forget the day you are told your child has a one in a million rare disorder: the overwhelming fear of isolation, fear of what the future holds…
A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…
"When only 100 people in the world share your child’s specific gene mutation, it is hard to find anyone who understands, including doctors." Rare diseases are at a disadvantage when…
When Julie Francisco was running on her 55th birthday, she could not ignore the pain in her lower abdomen and how “off" and slow her body felt. After a…
According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
According to a story from EurekAlert!, a team of scientists affiliated with the UCLA Jonsson Comprehensive Cancer Center have been conducting research that has revealed a new potential therapeutic approach…
According to a story from VC Reporter, Dr. Sabine Hazan-Steinberg is a true believer in the medical capabilities of fecal microbial transplant (FMT). Despite the fact that this procedure has…
According to a story from Parkinson's News Today, both doctors and patients may delay treatment of Parkinson's disease because of concerns about side effects. The most common treatment for the…
According to a story from BioPortfolio, the biopharmaceutical company AVEO Pharmaceuticals and the lung cancer diagnostic company Biodesix recently released the results from the Phase Ib clinical trial testing a…
The FDA has just announced that they've approved Mayzent for the treatment of relapsing multiple sclerosis (MS). Multiple Sclerosis Multiple sclerosis is a neurological autoimmune disease. It causes a wide…
The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company's application to…
According to a story from Market Screener, the biopharmaceutical company Imbrium Therapeutics LP recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation for the…
.jpg)
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the FDA based on interim results of the clinical trial ADMIRAL, according to an article in EurekAlert for Science News. A…
According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…
According to a story from andreeger.com, gene therapy, a type of medical treatment in which genes are modified in order to treat disease, has become a hot button topic in…
According to a story from businesswire.com, the biopharmaceutical company PellePharm, Inc. recently announced that it has dosed the first patient in the company's Phase 3 clinical trial. This trial is…
According to a story from EurekAlert!, a recent research study has found that a two part immunotherapy combination treatment has the potential to be a useful treatment for high-grade neuroendocrine…
According to a story from Science & Technology Research News, at team of researchers led by Kenneth S. Kosik, who is affiliated with UC Santa Barbara, have made a discovery…
Recently Dr. Janet Maynard, who currently is the director of the US Food and Drug Administration's (FDA) Office of Orphan Drugs Development, wrote an article in which she discusses the…
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.
