This Family is Fundraising After Two Rett Syndrome Diagnoses
The Munson family is fundraising to make changes to their home, as these adaptations will make life easier for their two children Oscar and his older sister Mary. This is…
The Munson family is fundraising to make changes to their home, as these adaptations will make life easier for their two children Oscar and his older sister Mary. This is…
Endometrial cancer is the most common form of cancer that develops in the uterus, and there is currently a need for more treatment options, especially when it comes to the…
The coronal suture is located at the top of the head, and it is responsible for separating the two parietal bones within the frontal bone of the skull. It is…
Tracey Shephard and her partner David Luffingham lost their daughter to meningitis and are now sharing their story as a warning to parents and medical professionals throughout England. The 11-year-old,…
Many little girls dream of being princesses, and Michele Kingsland is no different. Well, she is a little different, because her wish was actually granted. The Make-A-Wish Foundation crowned Michele…
The CDC has issued a warning regarding melioidosis, a rare bacterial infection that is typically found in tropical climates, as it has reached the United States. At the time of…
Kenneth Mitchell is an actor who has worked on the "Star Trek" franchise since 2017. He's played the Klingon Kol, the scientist Aurellio, Tenavik and Kol-Sha, alongside two other Klingons.…
A 15-year-old from Sydney, Australia has recently passed away after a battle with pneumococcal meningitis and a positive COVID-19 test. Osama Suduh was admitted to Sydney's Children's Hospital with a…
A small study has recently discovered that sweet marjoram tea helps to lower depression and non-motor symptoms when taken daily in combination with the standard antiparkinsonian medication. While larger studies…
Rare diseases often need extra support when it comes to research and drug development. Luckily, MMS Holdings Inc. (MMS) plans to provide some with their new support program. The program,…
Nivolumab has recently received an additional indication for the treatment of urothelial carcinoma with a high risk for recurrence following radical resection, a form of bladder cancer. This decision came…
Chimeric antigen receptor (CAR) T-cell therapy has proven to be a successful therapy for a number of conditions; however, we do not see the same success in follicular lymphoma and…
Recent data published in Gynecologic Oncology has confirmed the safety of olaparib for the treatment of advanced, BRCA-mutated, newly diagnosed ovarian cancer. This data came from the Phase 3 SOLO1 trial,…
Patient Worthy was pleased to serve on the advisory committee for the development of this new resource. Caring for a child with a rare disease can be scary and confusing;…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
Merck has just received FDA approval for their recently acquired drug Welireg. This HIF-2α blocker is indicated for von Hippel-Lindau disease (VHL). This rare disease gives affected individuals a higher…
AstraZeneca is ending a phase 3 trial of Ultomiris, which was being evaluated as a treatment for amyotrophic lateral sclerosis (ALS). The pharmaceutical company acquired the C5 inhibitor after buying…
Taylor Wilson, a woman from Buffalo, is raising money for hemophagocytic lymphohistiocytosis (HLH) in honor of her late friend Paige Casillo. The two have known each other since they were…
Developing therapies for rare diseases can be difficult, and it often requires effort from many different players. Recently, AllStripes has made large steps forward in rare disease research by securing…
The FDA has recently accepted Genentech's Biologics License Application (BLA) under priority review. This application was for faricimab, a bispecific antibody meant to treat ocular disorders, such as wet age-related…
Holden and Elliot have felt a surge of support from their community recently, as their preschool came together to celebrate them and show their love. Both boys were born with…
We're still learning about COVID-19 and its lasting effects, and we'll continue to do so for a long time. Researchers at Mount Sinai have been making progress, specifically with the…
It is important that we understand how medical conditions impact everyone, as knowledge helps with future research, drug development, and more. Unfortunately, medical research is not always inclusive. Because of…
While extremely rare, it is important to be prepared in case of vaccine-induced immune thrombocytopenia and thrombosis (VITT). This condition only occurs in 14.2 of every million COVID-19 vaccine doses,…
Rachel Ablondi recently wrote an article expressing her journey and thoughts on her son's life with Lennox-Gastaut syndrome (LGS) in an effort to get a message across to doctors. While…