Written by Dr. Lauren Steddum So far in 2021, more than 54,000 people have been diagnosed with oral cavity and oropharyngeal cancer and nearly 11,000 people have died of the…
Many doctors have thought in the past that age should be an automatic ineligibility factor for multiple myeloma patient transplants. However, Dr. Langren, Dr. Gasparetto, Dr. Landgren, Dr. Hofmeister, and…
On October 30-31, 2021, the Periodic Paralysis Association (PPA) hosted the Periodic Paralysis Conference in Orlando, FL. This event was an opportunity for patients to meet one another, share experiences,…
The University of Pittsburgh School of Medicine along with Prairie View A&M University, have recently uncovered that there are some cancer drugs which could be used as an effective treatment…
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…
LEXEO Therapeutics and FUJIFILM Diosynth Biotechnologies have just announced their collaborative partnership. The aim is to develop and ultimately manufacture the AAV-mediated gene therapies developed by LEXEO. LEXEO LEXEO is…
A new article has been published in Neurology and Therapy which specifically discusses the lived experiences of patients with myasthenia gravis (MG). The most notable thing about this article is that…
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…
Three doctors recently got together to discuss how they manage care for their sickle cell disease (SCD) patients. As a part of this conversation, they discussed how they combat systemic…
According to a story from echolive.ie, ten year old Adam Terry from Cork, Ireland, has directly appealed to Micheal Martin, the Taoiseach (or prime minister) of the country in order…
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…
According to a recent article from UTHealth Houston, a five-year-old suffering from a severe case of spina bifida has brought together a number of doctors to give her the…
In a news release from September 30, 2021, biopharmaceutical company Arrowhead Pharmaceuticals ("Arrowhead") shared that it had initiated its Phase 2b AROAPOC3-2002 clinical trial and that the first patient…
Dr. Ed Neilan, who is the Chief Medical and Scientific Officer of the National Organization for Rare Disorders (NORD), recently commented that there are approximately more than seven thousand…
October 23 is recognized each year as Kabuki Syndrome Awareness Day, a time to help spread awareness about this syndrome among the general public and in the medical field. Kabuki…
According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria Paroxysmal nocturnal hemoglobinuria (PNH)…
Data has recently been announced from a study investigating the efficacy of a new therapy called Alpelisib, for the treatment of PIK3CA-related overgrowth spectrum (PROS). The study was called EPIK-P1.…
CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments. CurePSP gives these grants twice every year.…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
On September 21, 2021, Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. The focus of this webinar was newborn screening in recognition of Newborn Screening Awareness Month, which is…
Funding is a problem that many rare diseases face when it comes to research. Cholangiocarcinoma, also known as bile duct cancer, is no exception. Because of this, it is often…
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…
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 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…
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