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…
Twins have always been a thing that has mystified the world, intrigued people, and definitely a curiosity. Fraternal, Identical, and everything in between – multiple births are a source of…
RARE on the Road 2021: Rare Disease Leadership Tour Virtual Training and Networking: Florida May 11, 2021 Global Genes and the EveryLife Foundation for Rare Diseases have worked together since…
Sanath Ramesh, a software engineer, created the nonprofit Open Treatments after his son was born with an extremely rare condition without any viable treatment options. Raghav, now two and a…
Since its inception, the Clinical Data Interchange Standards Consortium (CDISC) has worked to advance data standards to the highest possible quality. These standards are then used to make data more…
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…
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…
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