Canada’s unemployment rate is at a record low. Industries have recovered 126% of the jobs lost by the pandemic. On the health front, scientific knowledge and the ability to…
For patients with refractory or relapsed KMT2A rearrangement or NPM1-mutations, the AUGMENT 101 study of the investigational menin inhibitor revumenib yielded impressive results. According to an article in MedicalXpress,…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
There is no cure for cystic fibrosis. That is the reason the Cystic Fibrosis Research Institute (CFRI) is very vocal and makes it clear that cooperation is needed from the…
Throughout the globe, there are many countries in which rare disease awareness and support are still lacking. While India has made some strides in improving care for people within…
When Kristen Moore’s son Jackson was first diagnosed with tuberous sclerosis complex (TSC), she decided to translate her emotions into action. As WSOC-TV reports, she dove into education, awareness,…
According to a story from Healio, the US Food and Drug Administration (FDA) has recently updated the indication for the drug teprotumumab (marketed as Tepezza). Approved for the treatment of…
After becoming a de facto football star while playing at LSU, catching 52 passes for a total 629 yards, tight end (TE) Foster Moreau was drafted by the Las…
In July 2022, Patient Worthy reported on data from the Phase 3 REAL 4 clinical trial. During the trial, researchers compared the safety, efficacy, and tolerability of Sogroya (somapacitan-beco)…
As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
According to a story from Endpoints News, the drug developer Incyte has opted to put an end to its phase III clinical trial evaluating parsaclisib, an orally available PI3K inhibitor,…
Dr. Alan Grant (portrayed by Sam Neill) began Jurassic Park with a distaste for computers and children, intent only on developing his paleontological prowess. By the end of the…
According to a story from MedCity News, the drug company Eli Lilly was anticipating the approval and launch of its drug mirikizumab as a treatment for ulcerative colitis. However, the…
The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
According to a story from wmur.com, a 12-year-old boy named Grady Smith from Salem, NH lives with adrenoleukodystrophy, a rare disease. Grady is a big fan of basketball (his favorite…
In February 2023, I spoke with Dr. Allen Davidoff of XORTX Therapeutics Inc. (“XORTX”) about the company’s commitment to developing therapies for people living with progressive kidney disease. We…
According to a story from BioPharma Dive, the drug company AbbVie has recently announced its decision to withdraw two different approvals for its cancer therapy Imbruvica. The approvals were withdrawn…
STAT NEWS recently published an op-ed with a provocative question: How do we tell the difference between legitimate mental health tech and snake oil? Looking back to the early…
On April 23, 2023, people around the globe will celebrate Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day. April 23 marks the 17th anniversary of when scientists shared that they had…
On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
The International Association of Fire Fighters (IAFF) shared in March 2023 that firefighters in Ontario would now have presumptive pancreatic cancer and thyroid cancer coverage. Presumptive coverage means that…
Have you ever heard of the Orphan Drug Act? This Act helped stimulate and advance the development of, and research into, therapeutics for individuals living with rare or underserved conditions. In…
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