Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
Whitney Dafoe was an aspiring photographer who backpacked across the world. However, he always would return from his trips extremely exhausted. He would feel weak and he didn't know why.…
Severe Combined Immunodeficiency Severe combined immunodeficiency (SCID) is a group of diseases caused by mutations that affect how the immune system works to fight infections. These anomalies are usually passed…
2004 The pain in my right hip had periods of being intense. When the hip became unstable, I decided to visit my ortho. He took one look at it and…
1967 In 1967 my ortho said "this has to stop." I would just get out of traction; then a cast; heal; then be able to walk for 2 months and…
I was born in 1960 in Chicago, IL and they knew something was wrong seconds after my birth; what, they had no idea. My mother (I was her 3rd child)…
A reader named Fiorella, recently reached out in response to an article we wrote entitled: A Common Parasite With An Uncommon Name: Toxoplasma Gondii is Associated with Brain Cancer. This…
Researchers from the University of Barcelona have recently found a new biomarker which may be able to diagnose Parkinson's disease sooner. This biomarker is a protein fragment called GPR37. It…
Around 30% of all patients with relapsing-remitting multiple sclerosis (RRMS) experience some type of cognitive impairment or decline at the time of diagnosis. Around 50% of patients experience some of…
Primary Sclerosing Cholangitis Primary sclerosing cholangitis (PSC) is a rare condition which causes inflammation within the bile ducts of the liver. It can lead to cirrhosis of the liver or…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
By May 25th, 2021 the FDA will announce their decision regarding approval of Opdivo used in combination with chemotherapy for gastrointestinal cancers. Back in December, Priority Review was granted to…
Aeglea Biotherapeutics has just announced a new collaboration with Immedica to bring pegzilarginase to other countries outside of the United States. They have created a license and supply agreement to…
Rare Disease Day each year on February 28th provides a time for the rare disease community to reflect on the progress of the year, the continued gaps in rare disease…
A Phase 3 clinical trial run by Pharnext for Charcot-Marie-Tooth disease Type 1A (CMT1A) has just been awarded 13.3 million dollars. $7.2 million comes from existing shareholders and $6 million comes…
Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
COVID-19 has caused long-term, ongoing symptoms for some patients. Up until recently, these individuals had been called "covid long haulers." Anthony Fauci, during a White House briefing, has recently changed…
.png)
Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…
Kimberly Warner Kimberly Warner was diagnosed with mal de débarquement syndrome (MdDS) after enduring a traumatic biking accident in May of 2014. The accident caused her to break her pelvis…
Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been…
A recent study has documented the differences between two rare forms of childhood brain inflammation. The two similarly presenting conditions are often confused for one another. A better understanding of…
Mike Jackson's young daughter Evelyn was diagnosed with Batten disease after a neurologist suggested a genetic test which the family could take for free. John Barclay's daughter River passed away…
A recent study published in Cell Reports has uncovered a mechanism where germs living within the lungs of cystic fibrosis (CF) patients produce a type of carbohydrate slime that minimizes the efficacy…
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.
