Albert Edge and Yushi Hayashi recently led a research team which uncovered the specific mechanism which can cause deafness in Norrie disease, a rare condition. These findings were recently published…
On October 27, 2021, the National Organization for Rare Disorders (NORD) hosted an informational webinar titled "Navigating Diversity, Equity, and Inclusion (DEI) in Rare Disease Non-Profits." This webinar was the…
Access Opportunities for Rare Disease Patients At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with…
According to a press release from Ikena Oncology, the FDA has recently accepted an Investigational New Drug (IND) application for Ikena's investigational drug candidate, IK-930. This TEAD inhibitor is intended…
Written by Rod Cisneros Like many people, I keep a daily journal. I use it to document my everyday thoughts and I even include advice for family members who might…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
A recent article in NewScientist describes Borrelia burgdorferi as a bacterium that is the cause of Lyme disease (borreliosis). The trail of infection begins with wild mice that harbor the…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
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…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
A recent article in Science Alert reports on early but positive results from a first-in-human Phase 1 clinical trial of ProTide NUC-7738. About the Drug Researchers at Oxford University partnered…
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…
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…
An October 2021 report published in the Globe and Newswire carried an announcement by Fabio Chianelli, CEO of the biotech company PharmaTher Holdings. The FDA approved orphan drug status for…
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…
Cooking and eating together is a great way to build closer bonds and spend time with those we love. In Malaysia, it's a large part of the culture, as there…
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…
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