On October 27, 2021, the National Organization for Rare Disorders (NORD) hosted an informational webinar titled "Navigating Diversity, Equity, and Inclusion (DEI) in Rare Disease Non-Profits." This webinar was the…
Access Opportunities for Rare Disease Patients At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
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…
Aurion Biotech has just released the results from their clinical trial called IOTA. This trial was conducted by researchers in Japan and demonstrated that corneal endothelial cell therapy was able…
In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
CRISPR (clustered regularly interspaced short palindromic repeats) was discovered over a decade ago by Emmanuelle Charpentier and Jennifer Doudna, two scientists who recently shared the Nobel Prize. Scientists using…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
Early diagnosis is critical in many rare diseases, as earlier intervention leads to better treatment outcomes. Now, diagnosis is becoming easier and quicker through the use of artificial intelligence (AI).…
The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came…
According to a recent article in Yahoo News, unless treatment is discovered or a cure is found for a disorder called IRF2BPL, these young children will lose the ability to…
nivolumab Read the source article at healio.com
Health officials in Long Island, New York are investigating ten reported cases of Legionnaires' disease, which is a severe form of pneumonia. While medical teams are searching the area for…
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…
Throughout my daughter Kelley’s 45-year life with a rare disease, there were numerous times that we needed to either call 911 or take her to the Emergency Department at a…
Climate change is an issue that has seen a lot of activism from the younger generations, as they will have to grow up and handle more of the burdens it…
On October 25, 2021, Rare Disease Legislative Advocates (RDLA) held a virtual briefing for the Rare Disease Congressional Caucus. The subject of this briefing was newborn screening and ongoing discussions…
On October 7th, 2021, Patient Worthy took part in RARESummit21, a virtual event organized by the Cambridge Rare Disease Network (CRDN). "...a flagship CRDN event gathering over 300 great minds…
Collaboration can be extremely important when it comes to developing treatments, especially for rare disorders. Sharing information, technology, and expertise can lead to therapies that otherwise might not have seen…
Every game, the men of Northwestern Football leave the tunnel to a sign with an important reminder: “trust yourself.” Each player touches that sign before they head to the field,…
Rare Diseases South Africa (RDSA) and The Rare Diseases Access Initiative (RDAI) have recently come together to talk about the issues that those living with rare diseases in South Africa…
Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
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…
A new partnership has been formed between Global Genes and the Rare Disease Diversity Coalition (RDDC) - created by the Black Women's Health Imperative - in an effort to increase…
A lack of awareness is a problem that many rare diseases face. Small patient populations, limited research, and other factors all play a role in this issue. Luckily, there are…
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