September is Dystonia Awareness Month so I want to share a little about it and how it impacts those of us who live with it. I have to be honest…
Often when a cure for a specific rare disease has yet to be developed, patients have enrolled in clinical trials with positive results. However, FDA-approved trials have developed a reputation…
The Family Heart Foundation has a Mission. There is so much more to accomplish and you can help prevent heart attacks by spreading awareness. This could save future generations by…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
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…
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…
Unfortunately, the drug development process can be difficult, especially in the rare disease sphere. Many drug developers choose to focus on larger disease states, as these are typically more…
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…
Before you read, make sure you check out Parts 1 and 2 of our interview. In Part 1, we discussed what Wegener's granulomatosis (GPA) is, Anna's diagnostic journey, and her first episode of symptoms.…
Many people laud the accomplishments of clinical trials – and for, in some cases, good reason. Clinical trials have been crucial in identifying and developing therapeutic options for patients to…
It’s no secret that being a part of the chronic illness and/or rare disease community can sometimes be lonely and isolating. Many people, while empathetic to the challenges faced within…
Lee Neal age 38 and his wife Jessica age 35 were on holiday when they noticed a slight swelling in their toddler’s eye. Sixteen months later, their baby died. Now…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
Six years after receiving a lung transplant a young woman 21 years of age noticed an article in Reforestemos Patagonia © about another young woman who was waiting for a…
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…
Before you read on, make sure you take a look at Pt. 1 of our interview, which goes over what PGAP3 is, Lucy's story, and the reasons behind why Zach and Geri…
Targeted Oncology recently published an article describing a group of T cell malignancies associated with blood disorders (hematologic cancers). Currently, a high rate of patient deaths and relapses are occurring…
Inside Precision Medicine recently featured an article describing a study that was published in the European journal Diabetologia conducted by a team of Chinese and Swedish researchers. The study suggested…
On July 27, 2022, the National Organization for Rare Disorders (NORD) hosted an online webinar titled "Drug Development for Rare Diseases: A Community Conversation." This program was designed to help…
The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this month is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…
Rare Disease Diagnoses Get a Boost from Scientists’ New guidelines Professor Dame Sue Hill, Chief Scientific Officer of NHS England, recently interviewed with MedicalXpress citing the importance of unraveling the mystery…
On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community…
When Ryker Colón was first born, his parents Eddie and Brandi noticed something potentially concerning. Their young son had a chronic cough - and nobody could seem to figure out…
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…
Written By Ashely Winslow, PhD, President & Chief Scientific Officer of Odylia Therapeutics One of the biggest issues impacting many rare disease communities is the availability of approved treatments. While…
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