According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
Caregivers come from all walks of life but they share one common goal- taking care of someone they love who cannot fully take care of themselves. This may mean helping…
Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It's a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can…
Tiffany Rowe was 15 when she accomplished her biggest childhood dream. She met Michael Jackson at a live concert and actually got to dance on stage with him. Over…
According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that…
"It's funny to say grief as it is not a bereavement but you kind of are grieving the loss of the child you thought you had and the future they…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
On November 26, 2018, the FDA approved a new autoinjector for individuals with severe rheumatoid arthritis (RA), giant cell arteritis, and polyarticular or systemic juvenile idiopathic arthritis (aged 2 and older). It's…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are thousands…
The US Food and Drug Administration recently announced the approval of the first biosimilar drug for patients with non-Hodgkin lymphoma in a press release. The drug is known as Truxima…
Garret Schuster was just six months old when he was diagnosed with hemophilia. He went to see a pediatrician to treat an ear infection but the doctor sent him for…
For many families, when their child is born with developmental delays, there may be weeks, months, or even years that go by without a diagnosis. During this time, parents…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are…
According to a story from Digital Journal, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that its investigational drug candidate ACE-083 was granted Fast Track designation from the US Food…
Lynch Syndrome Lynch syndrome is a genetic condition which makes an individual more susceptible to certain types of cancers. Specifically, it has been thought to increase risk for developing colorectal…
For the last 3 decades, the only treatment that has been available for hereditary transthyretin-meditated amyloidosis was a liver transplant. The condition is caused by abnormal transthyretin proteins. These proteins…
According to a story from BioPortfolio, Celgene Corporation and bluebird bio, Inc., have recently announced that enrollment for a pivotal Phase 2 study testing an experimental therapy for relapsed/refractory multiple…
According to a story from The Star Online, the government of Malaysia has allotted RM50 million of its 2019 budget for the treatment of numerous rare diseases, stunted growth, hepatitis…
In 2013 researchers discovered something interesting about the protein BH4. They found that by inhibiting the protein they could reduce patient's pain. A drug was created called Quartet, and it…
The Asia-Pacific Economic Cooperation (APEC) has 21 members including the United States, China, Canada, Australia, Peru, Russia, and Thailand to name a few. They work to encourage the interdependence of the…
The world is recognizing more every day just how important rare disease research is. Yes, rare diseases are rare but they affect millions of people worldwide. The more we can…
According to a story from tgen.org, a team of scientists from the Translational Genomics Research Institute (TGEN) have recently initiated a study that will test the effectiveness of liquid biopsy…
4D110 has just received Orphan Drug Designation for the treatment of Choroideremia (CHM). The phase 1 trial of the drug is expected to start in 2019. What is Chorioderemia? Chorioderemia is…
According to a story from the Farmington Daily Times, Dr. C. Frank Bennett, who grew up in Aztec, NM, and his collaborator Dr. Adrian Krainer, were both jointly awarded the…
According to a story from Business Wire, the drug developer Cycle Pharmaceuticals recently announced that is has completed its goal to obtain the necessary data regarding the stability of its…
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