Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…
We’ve all become a little more in tune to what’s good for our bodies, and what isn’t. Part of that is medical science finally being able to identify our bodily…
According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
According to a story from The ME Association, a recent debate that came to the floor of the UK parliament highlights the misinformation and deficits in care surrounding children with…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
According to a press release from X-Rx Inc., a privately-held American biotechnology company, the Food and Drug Administration has accepted the company's Investigational New Drug (IND) application for X-165, an…
deCODE Genetics, which is a research subsidiary of Amgen genomics based in Iceland, has recently created an extremely detailed genetic map. They wanted to uncover more about the human genetic…
According to a story from Renal & Urology News, a recent study found that monitoring of albuminuria, a condition in which the protein albumin appears in the urine, can help…
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…
There is a high unmet need to find more effective treatments for liver cancer. Currently, treatment options for patients often only increase life expectancy by 3 to 4 months. An accidental…
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.
