On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
RARE on the Road 2021: Rare Disease Leadership Tour Virtual Training and Networking: Nevada May 4, 2021 Global Genes and the EveryLife Foundation for Rare Diseases have worked together since…
World Orphan Drug Congress USA 2021 Free Virtual Event Online April 28, 2021 The Global Orphan Drug Conference and Expo Don't miss out on this global event featuring over 1,000…
According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company…
The Rare Disease Legislative Advocates (RDLA) held its monthly webinar on April 22, 2021. In this informational webinar, the organization featured several speakers who provided updates on some of the…
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A decade ago, Rob Long's dreams were centered solely on football. He was looking forward to the NFL draft after a successful college career at Syracuse University, but unfortunately, a…
In a previous article, I talked about the importance of patient-led advocacy groups and how they can positively impact how a rare diagnosis is received and understood by patients. Do…
Scholarships are being provided to rare disease patients, thanks to the EveryLife Foundation for Rare Diseases. The scholarships are each worth $5,000 and are intended for those seeking an education…
For small patient groups, creating momentum towards rare disease solutions often seems futile. To tackle such expensive and extensive endeavors, rare communities find strength and hope by putting their minds…
Rare Mother Meetup 6:00 PM, PST The Rare Mother Meetup will take place on the second Wednesday of each month and is hosted by Rare Mother Ashley Kenny. The meetup…
Each year, researchers make new strides into genetic research, including the discovery of new genetic disorders. According to Medical XPress, researchers from the University of Portsmouth and the University of…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
On April 2nd, NFL scouts made their way to Eugene, Oregon to check out some of college football's most talented players. They watched as athletes completed the vertical jump, 40-yard…
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…
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…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
In a recent contribution to MSN, Dr. Christopher Austin, director of the NIH Advancing Translational Sciences, compared the medical community’s record-breaking approach to the COVID-19 pandemic with current efforts…
Rare disease patients face a number of obstacles when it comes to getting the proper diagnosis and treatment. There's a lot of work being done to address these problems, some…
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…
MSN News recently interviewed Dr. Laxmikant Palo, with the conversation focusing on the situations of people living with rare diseases in India and how to improve them. According to Dr.…
Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
The Rare Sibling Experience March 25, 2021 Rare disease impacts extend beyond the patient themselves and can have a profound effect on families. Siblings of rare patients are placed in…
Journaling and Resilience Workshop Pamela Alma Weymouth will lead an hour long session of reflection and journaling. Learn self-acceptance and gratitude while writing about your week to cultivate resilience and…
No longer the new player on the team, next-generation sequencing (NGS) is well established as having an influence in clinical care. In recent years, NGS has been responsible for many…
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…
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