An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
The other day I read a post in a dystonia support group that is similar to something I see and hear very often. I’m sure you have as well, and…
Genethon is a non-profit organization committed to the design and development of gene therapies for rare diseases. This unique company is a pioneer in identifying genes relating to genetic…
Having lived with chronic pain and involuntary muscle contractions from a neurological movement disorder called dystonia since 2001, I felt defeated many times. But I am still here battling every day… and…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, Alex discusses the multi-year diagnostic odyssey to discover that Raymond has VAMP2. Today, we talk…
On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government's budget for the year, so naturally it includes…
Do you wish to be heard? Recently PR Newswire announced that on the 28th of February 2023, NORD and the worldwide community of rare disorders will be raising awareness…
To read part 1 of Nikole's Holiday Confession, click here. I feel guilty when my limitations force others to have to alter their holiday expectations. Like, how do I tell…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Pumpkin pie, ginger-bread cookies, candy canes, and chocolates. Oh what a sweet season! A few nights ago my kids and I spent a few hours crouched over the wafting smells…
Written by Lisa Ann Krutzik An inexpensive and extremely valuable gift you can give your older teen or adult child this holiday season... A letter from you. I spend a…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
A new awareness campaign called RKD & Me has recently kicked off with the goal of spreading awareness of rare kidney disease (RKD). The project is a collaboration between the…
The NIH’s All of Us Program is reaching out to those who have been underrepresented in medical research. The project has been heralded as a “major milestone.” In addition,…
Chronic pain reminds me of a bully that picks on kids in the schoolyard every day. Like the bully, chronic pain does not follow any rules and after multiple beatings,…
Before you read, don't forget to check out Part 1 of our interview! In Part 1, we discuss Ronya's story, genetic counseling, and why she chose to launch DNA in Color.…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
On December 8, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
When we look at statistics that show thirty million people in the U.S. has one of seven thousand rare diseases, they may not seem rare. However, when we realize that…
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