By Danielle Bradshaw from In The Cloud Copy Pompe disease is the lack of a specific protein within the body that helps break down glycogen - a complex sugar that…
According to a story from mdmag.com, a recent proof-of-concept style study suggests that a certain machine learning tool could be of benefit to patients living with the rare condition alkaptonuria.…
The term ‘failure’ may no longer apply to cancer vaccines according to a recent article in Marketwatch. Researchers have endured over four decades of frustration since the first two…
As originally reported in the Cincinnati Children's, Brittney writes how she came into the world of rare diseases when her and her husband Michael decided to adopt a baby who…
As reported in Creaky Joints and Healing Hugs Haven; the media has been overtaken by coronavirus on the move. Rare disease patients need to know what that means and what…
An issue with current treatments for Parkinson's disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
Patient confidentiality is often seen as an absolute in the medical world. Many people believe that it should never be broken. Cases do exist that challenge this thought however, where…
According to a recent article in Healio, the National Institute of Health states that one-third of strokes occurring for the first time to people under fifty can be linked to…
Shelley Simmonds recently attended the Festival of Genomics, the largest genomics event in the United Kingdom that is quickly growing into the biggest event in the world. It involves…
Primary sclerosing cholangitis (PSC) is a chronic liver disorder that typically affects males over the age of 30. That is why Di'Jon Thomas was so surprised to be diagnosed with…
1 in 10 people suffer from a rare disease, but when I talk about writing for Patient Worthy, it’s usually the first time people have had a conversation or really…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
Ashley and Chris Numbers are the parents of Alayna and Michael. Soon after their wedding they noticed that Alayna was covered in bruises, which prompted them to take her to…
Sarclisa was recently approved for the treatment of multiple myeloma when taken with pomalidomide and dexamethasone. The FDA indicated that this treatment is for those who have been treated with…
According to a story from Sixth Tone, life for the Lu family of Yuncheng, China, has never been easy. Life in the region has slowed thanks to the coronavirus, but…
Austin Jackson is a star football player at the University of Southern California. His entire life consists of football, whether that is training for it, watching it, or playing it.…
This year, the entire Patient Worthy team traveled up to Washington, DC for Rare Disease Week 2020. This event is always a big one for the rare disease patient community.…
Prevail Therapeutics has released an update on their Parkinson's disease treatment, PR001. They are currently dosing participants, and enrollment remains open. As of now, the study remains on track. Results…
Susan Mann was seven years old when she was diagnosed as having Hashimoto’s thyroiditis, an autoimmune disease that affects the thyroid. According to a recent article in the online…
As originally reported in Biospace, two research networks dedicated to forming bridges between rare disease research around the world will share their niches to promote discovery for rare diseases. TriNetX…
Todd Mercer, a fifty-two year old Michigan resident and father of two, recently called into STAT’S Podcast The Readout LOUD to discuss the difficulty he has had getting enrolled…
The European Medicines Agency has recently released their positive opinion of ALUNBRIG, which is a treatment for anaplastic lymphoma kinase+ (ALK+) non-small cell lung cancer (NSCLC). They have recommended this…
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