According to a story from Benzinga, the drug developers AstraZeneca and Merck have recently announced that the US Food and Drug Administration (FDA) has decided to accept a supplemental New…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…
A $1.4 million dollar donation to the University of Massachusetts Medical School will help to fund research into a gene therapy for Tay-Sachs disease as it transitions from pre-clinical to…
The annual Global Genes Rare Patient Advocacy Summit is wonderful place for families affected by rare disease to come together and interact face to face with advocacy leaders in addition…
Some experiences are seen through rose-colored glasses while others are hazy or ambiguous. Many, we try desperately to remember while others we we wish we could forget. And a special…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
I hope that you know a wonderful doctor. The type who listens wholly, respects you, validates your concerns and opinions, and goes above and beyond to make sure you get…
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
According to a story from Scleroderma News, a recent study found that men were more likely to have severe scleroderma early in their diagnosis in comparison to women. This is…
Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the…
A recent study has looked into the use of video visits with patients by clinicians in Sweden, to try to find out how doctors decide which patients to offer them…
As we age, we become more at risk for certain conditions. For instance, osteoporosis, atrial fibrillation, certain cancers, and heart disease, are more common in older individuals. These conditions pose…
According to a story from NRI Pulse, Kiran and Anju have been forced to watch over the past year as their daughter Aadya's physical and mental condition declined. After serious…
According to a story from BioSpace, the biotechnology company Partner Therapeutics, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company's…
According to a story from Hematology Times, an experimental CAR-T cell therapy called P-BCMA-101 was recently granted regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration.…
According to a story from Futurity, a recent study has identified a protein that plays a central role in the onset of a rare cancer called subcutaneous panniculitis T lymphoma…
In the movie "Dallas Buyers Club" the main character, Ron Woodroof, contracts HIV/AIDs and starts getting drugs from an organization that imports medicine from Mexico. Similarly, many London patients are…
In a recent news article, the Northwestern Medicine Feinberg School of Medicine has shared details about an experimental device that may have the potential to dramatically improve the treatment of…
According to a story from prweb.com, the Neuromuscular Disease Foundation (NDF) recently announced that is has earned a $2.5 million challenge grant. This massive grant will go towards funding the…
An investigational new drug for bronchopulmonary dysplasia, called CF-MEV-132, has completed pre-clinical in-vivo testing. According to The Cell Factory, who are developing the drug, the potential treatment is now ready…
According to a story from EurekAlert!, a recent study from the Children's National Health System has found that some cystic fibrosis patients are not getting sufficient doses of antibiotics in…
According to a story from statnews.com, Ella Balasa, who has cystic fibrosis, has been seeking to join a clinical trial in hopes of finding a new treatment that can help…
This is part two of Morgan's story. Read the first half here. I was taken off of work and school for three months to monitor my condition. I had to drop…
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