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…
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.
