GO-ALIVE GO-ALIVE was a Phase 3 clinical trial examining the safety and the efficacy of Simponi Aria in ankylosing spondylitis (AS). Simponi was first approved by the FDA in 2009.…
According to a publication from the University of Edinburgh, a new study found a set of 37 genes that were highly expressed in breast cancer tumour-associated macrophages (or TAMs -…
According to a publication from EurekAlert, a recent study suggests a common, typically symptomless form of the herpes virus called cytomegalovirus may cause faster disease progression in cystic fibrosis (CF)…
According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe in a phase 1/2 clinical…
According to a press release from New Jersey-headquartered ADMA Biologics, the FDA has approved the company’s primary humoral immunodeficiency disease (PIDD) drug Asceniv for the American market. The company expects…
According to an article at HealthEuropa, including patients and their families as an important component of cystic fibrosis research is a requirement when developing future treatments for the disease. That's…
According to an article at ScienceDaily, a combination of immunotherapy and chemotherapy therapies caused pancreatic cancer tumors to shrink in the majority of patients in a phase 1b clinical trial.…
LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug…
.jpg)
An encouraging article recently published in Sickle Cell Anemia News announced that due to the urging of pharmaceutical companies and patient advocacy groups, the FDA has produced an updated draft…
According to a publication from Live Science, researchers from around the world are working on a way to identify cancer in the body more efficiently and reliably. One way scientists…
Females vs. Males with Fabry Fabry disease is an X-linked condition caused by a malfunctioning GLA gene. It was long believed to only affect men. Since women have two X…
Any fans of RuPaul Drag Race or the art of drag itself? Raise your manicured hand! Well, regardless of your answer - watch how the world of drag and rare…
GT Biopharma has just announced that their Phase 1 clinical trial for Mastocytosis, Myelodysplastic Syndrome (MDS), and Acute Myeloid Leukemia (AML) has been authorized to begin. Although this investigation had previously…
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…
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.
