The 59th Annual European Society for Paediatric Endocrinology (ESPE) Meeting is taking place virtually from September 22-26, 2021. During the Meeting, researchers are discussing best practices and treatments, as well…
COVID-19 has brought attention to a rare condition that has been flying under the radar for years: chronic fatigue syndrome (CFS). With more attention, people are more willing to share…
According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…
There are inequities when it comes to race in the medical sphere, just as there are in every other facet of life. One specific issue pertains to clinical trials, as…
It's no secret that mosquitos can carry a variety of viruses and diseases: Zika virus, malaria, Chikungunya, and even the West Nile virus. However, Pennsylvania authorities recently detected another mosquito-borne…
Medical research drives a huge amount of our understanding around rare diseases. Recently, researchers from Brown University performed a study to deepen their understanding of a rare, X-linked genetic disorder…
The FDA has recently approved Skytrofa as a treatment for children at least one year of age who are facing growth failure as a result of an inadequate secretion of…
Within the United States, Orphan Drug designation is granted by the FDA to facilitate the development and review of drugs or biologics intended to treat rare conditions. In this…
When you think of effective fundraisers, do you think of pickleball? Well, a Kansas community did just that. According to a video and transcript on KMBC News 9, his community…
From certain partnerships can emerge not only a better understanding of rare diseases but potential therapeutic options. As described by BioProcess International, a nonprofit organization called the Institute for Life…
Treatment for patients with GM2 Gangliosidosis - which consists of Tay-Sachs disease and Sandhoff disease - has often been symptomatic and supportive. However, a new therapy shows promise in benefiting…
Many rare diseases face issues when it comes to awareness, we know this. Low patient populations, minimal research, and little knowledge all contribute to this problem. One father aims to…
Pregnancy can be stressful enough as is without the looming threat of COVID-19, a novel coronavirus which has played a huge role in our lives since the beginning of 2020.…
The FDA has recently approved a new diagnostic tool to help determine which IDH1-mutated cholangiocarcinoma patients may be a good fit for the novel IDH1 inhibitor called ivosidenib. The new…
In the past, Exelixis' drug CABOMETYX (cabozantinib) has been approved for use in a variety of patients. For example, the treatment is approved for patients with renal cell carcinoma (RCC)…
A recent study has investigated the association between cardiovascular disease risk factors and fat distribution. Researchers used linear regression analyses and adjusted for insulin sensitivity and other factors. Previous research…
According to a recent article at Medical XPress, the number of intensive care units around the world overwhelmed by critically ill patients suffering from COVID-19 leaves organ transplant patients with…
Recently, hundreds of people celebrated the 12th Annual Fara 5k in Providence, Rhode Island. This race was organized over a decade ago in an effort to raise money for Friedreich…
An estimated 30% of patients with psoriasis go on to develop psoriatic arthritis (PsA), a type of inflammatory arthritis that can affect joints throughout the body. One particular spot which…
Angela Singletary is the proud mother of three children: Aidan, DL, and Angela. One of them sticks out from the rest - literally. DL is a foot and a half…
According to a story from news.cision.com, the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for CS1, an investigational drug being developed by the…
After a case of the rare tick-borne illness, Powassan virus, was reported in Providence, Rhode Island in June, Patch released an article detailing the different diseases ticks can carry, how…
According to a press release from Business Wire, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive opinion in regards to the…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
ABC News recently featured an article about Isabella Cristobal and Sergio Soto. They decided to get married shortly after learning that Sergio’s cancer had spread and he had only…
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