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…
Although many people in the United States face challenges when seeking healthcare services, new research has found that when seeking a diagnosis for a rare disease, the situation is…
The National Organization for Rare Disorders (NORD) recently held its Living Rare, Living Stronger Patient and Family Forum on June 26, 2022. This event took place both virtually and in-person…
According to a recent article, a man who developed terrifying symptoms following a stroke was found to have statin-associated immune-mediated necrotizing myopathy. Unexpected Symptoms After Stroke When her…
The novel program called BeginNGS™ was described in a recent article which has a goal of testing for five hundred disorders and several thousand infants. Rady Children's Institute 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…
Getting a rare disease diagnosis can be difficult and take a long time. This can be a problem, as time is something that those with progressive illnesses don't have much…
According to a recent article, a new study where SIOD patients had a dual immune/solid organ transplant yielded positive results with patients accepting their new kidneys without rejection. Schimke Immune-Osseous…
According to a recent press release from Biospace, the first cohort of the ReSPECT-LM trial has officially completed enrollment. This trial focuses on Rhenium-186 NanoLiposome (186RNL) as a treatment for…
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…
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