LymeDisease.org is one of the most distinguished nonprofits for Lyme disease in the United States. In 2015 they created MyLymeData in the effort to improve treatments for this disease. 10-20%…
According to a story from Yahoo Finance, the biopharmaceutical company Mallinckrodt recently released findings from an open label pilot study which tested the impact of Acthar Gel (corticotropin injection) as…
According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the…
According to a story from Healing Hugs Haven, the rapid spread of COVID-19 around the world has been getting a lot of airtime in the news lately, and for good…
AVROBIO has recently received the Orphan Drug designation for AVR-RD-04, a gene therapy for the treatment of cystinosis. This treatment takes the stem cells of those affected and modifies them…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
According to a story from Financial Buzz, the biopharmaceutical company Mereo BioPharma Group plc recently announced that it has successfully concluded its Type B End-of-Phase 2 meeting with the US…
In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…
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.…
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…
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…
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…
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…
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…
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