Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
St. Jude Children's Research Hospital has recently announced that its team of scientists have established a registry for pediatric melanoma. The goal is to use data from this registry for…
My nickname is Arti and I'm 11 years old. Because of my HAE diagnosis, I've been suffering from frequent and painful swellings since I was little. My life is divided…
Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it…
The month of July is recognized as Sarcoma Awareness Month, a time of the year for spreading awareness about sarcoma among the general public and in the medical community. An…
Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
In the past, arimoclomol has not always shown treatment promise. For example, the drug failed to meet primary or secondary endpoints in a clinical trial evaluating arimoclomol for patients with…
According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…
Unfortunately, many molecules which drive cancer development and growth are considered "undruggable." In short, this means that drugs are unable to bind to or address these molecules due to size,…
Even though my boyfriend's mom works at an eye doctor, I'm still kind of afraid to get eye exams - and I'm not alone. Unfortunately, most people do not receive frequent enough…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
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…
The European Hematology Association (EHA) held its 2021 Virtual Congress from June 9-17, 2021, though the insights will be available until mid-August 2021. During the Congress, researchers discussed new…
A new study, funded by the NIH and conducted by Vanderbilt University Medical Center (VUMC), is working to assess allergic reactions to COVID-19 vaccines. The trial will include individuals between…
Two new reports of Guillain-Barré syndrome (GBS) have been documented in response to the AstraZeneca COVID-19 vaccine. Previously, published papers have documented 4 cases in England and 7 cases in…
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
According to a recent article in Genetic Engineering and Biotechnology News, the blood-brain barrier (BBB) regulates the movement of molecules, cells, and ions between the brain and the blood. Tightly…
As reported in Fierce Pharma; BridgeBio has earned FDA approval for their new targeted cancer treatment, Truseltiq. The approval is the biotech company’s first ever approval for a cancer drug,…
We know that COVID-19 leaves some with long-lasting symptoms. In fact, we are beginning to discover the connection between the respiratory virus and chronic fatigue syndrome (CFS). Now, CFS patients…
Dr. Jerry Vockley, MD, PhD, who currently works as director of the Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh, was confounded. Through his research, Dr. Vockley…
Auto-Brewery Syndrome Auto-brewery syndrome is also known as gut fermentation syndrome. It occurs when fungi or bacteria within the gut produce ethanol by endogenous fermentation. Several different strains may be…
It's no secret that illicit substance use, or substance use disorders, can cause a variety of health effects, including increased risks for certain diseases. As Parkinson's News Today recently reported,…
In patients with rare or underserved conditions, it can sometimes be difficult to receive effective treatment. For example, chemotherapy, the standard-of-care for Langerhans cell histiocytosis (LCH), is only effective in…
Partnerships within the medical community play a huge role in advancing research and developing treatments. According to Charcot-Marie-Tooth News, a new partnership has formed between Dr. Alessandra Bolino, PhD, the…
Newborn screening is important to early diagnosis and intervention. With some conditions, every second counts. Catching a rare disease as soon as a child is born could lead to much…
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