In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
Sometimes, treatments used in conjunction with each other can confer benefits for patients. According to the Cancer Network, for example, treating platinum-resistant ovarian cancer using nemvaleukin alfa and pembrolizumab (Keytruda)…
Healx has just announced that they've been given Investigational New Drug FDA approval for HLX-0201 for Fragile X syndrome. This means they are able to move forward with their Phase 2a…
CRISPR (clustered regularly interspaced short palindromic repeats) was discovered over a decade ago by Emmanuelle Charpentier and Jennifer Doudna, two scientists who recently shared the Nobel Prize. Scientists using…
While there is no cure for patients with systemic scleroderma, there are some therapies to manage symptoms, such as medications, antibiotics, physical therapy, or even surgery. But according to Medical…
According to a recent article, surveying sewage proved to be the key to helping scientists predict new virus outbreaks. How It Works It may seem crazy that your feces could…
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…
Aurinia Pharmaceuticals Inc. plans to release the updated results from their continuation study of AURORA 2. This clinical trial is evaluating LUPKYNIS, also known as voclosporin, as a treatment for…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
According to a recent article, a mother was able to see her baby daughter clearly after being diagnosed with the rare Stargardt disease. Stargardt Disease Stargardt disease is an inherited…
Early diagnosis is critical in many rare diseases, as earlier intervention leads to better treatment outcomes. Now, diagnosis is becoming easier and quicker through the use of artificial intelligence (AI).…
According to a news release from biopharmaceutical company Clearside Biomedical, Inc. ("Clearside") and eye health business Bausch + Lomb, a revolutionary treatment called XIPERE (triamcinolone acetonide injectable suspension) was recently…
The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came…
When Shelby Sparks was just five months old, she began having grand mal seizures. Throughout her early years, her family just believed that their daughter had severe epilepsy. Then, when…
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…
For years, Debra Papagni Denmark worked to have New York State recognize Phelan-McDermid Syndrome after her son Drew, now 13, was diagnosed. A few years ago, Governor Andrew Cuomo heard…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Lyme disease is typically treated with a broad-spectrum antibiotic. Unfortunately, these can have just as many negative effects as they can positive. For instance, they can increase the chance that…
According to a recent article in Yahoo News, unless treatment is discovered or a cure is found for a disorder called IRF2BPL, these young children will lose the ability to…
According to a news article published by the Yale School of Medicine, Yale University researchers recently determined that autosomal dominant polycystic kidney disease (ADPKD) could potentially be reversed. Head researcher…
nivolumab Read the source article at healio.com
Also known as Whitmore's disease, melioidosis is a rare and deadly infectious disease. While rare in the United States, melioidosis is considered a public health crisis in northern Australia, Southeast…
November 1st marks Lennox-Gastaut syndrome (LGS) Awareness Day, which is dedicated to patients living with this severe epilepsy syndrome. The LGS Foundation has organized this day to celebrate the resiliency…
In August 2021 News Center Maine ran an article introducing former British army Major Chris Brannigan. Brannigan, forty-one years old, was prepared to do anything to help his daughter Hasti,…
Health officials in Long Island, New York are investigating ten reported cases of Legionnaires' disease, which is a severe form of pneumonia. While medical teams are searching the area for…
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