Mother and Son Receive Hemophilia B Diagnosis
In 2002, three-year-old Collin Johnson underwent a tonsillectomy. Just a couple of days later, after Collin woke up from a nap, Stormy noticed a large pool of blood on the…
In 2002, three-year-old Collin Johnson underwent a tonsillectomy. Just a couple of days later, after Collin woke up from a nap, Stormy noticed a large pool of blood on the…
In the medical world, it’s no secret that misdiagnoses happen. But these misdiagnoses can be costly; patients may miss out on earlier treatment and prevention, and misdiagnosis may correlate with…
Previous research presents strong evidence favoring shared decision-making after a medical diagnosis. One major improvement has been designing treatment plans that include a good deal of the patient’s goals and…
Did you know that November is considered to be Hyperhidrosis Awareness Month? This awareness campaign is intended to share information about hyperhidrosis (a medical condition causing excessive and uncontrolled sweating),…
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…
According to a story from PR Newswire, Immunic, Inc., a biopharmaceutical company prioritizing the development of oral immunology medicines for the treatment of chronic inflammatory and autoimmune disorders, has reported…
The loss of axons (nervous system wiring) has been responsible for various neurodegenerative diseases such as peripheral neuropathies, Parkinson’s disease, glaucoma, and amyotrophic lateral sclerosis (ALS). According to a…
Mahesh Karande, the President and CEO of Omega Therapeutics, is incredibly passionate about epigenetics. And it shows: in the work that he and his team do, in the therapies they…
In the ongoing search for prevention and a cure for Alzheimer’s Disease (AD), a study was conducted at Colorado’s Anschutz Medical Campus. The researchers found that memory and learning are…
Real World Evidence Note: This story was originally published by our partner organization TREND Community. You can check out the original publication here. When I was nine years old, I…
If you ask college basketball fans in Tennessee, they’ve probably heard of Zakai Zeigler. The 5’9 point guard, who originally hails from Long Island, now dominates the court as a…
So far, the Phase 2 EMPOWER-CSCC-1 clinical study has evaluated cemiplimab (also known as Libtayo) in individuals with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). Data from…
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…
According to the National Institute of Neurological Disorders and Stroke, chorea is defined as involuntary movements that are caused by a neurological disorder. It is one of a group of…
According to a press release from the US Food and Drug Administration (FDA), the agency recently approved a new treatment for multiple myeloma, a type of rare blood cancer. The…
In May 2022, 14-year-old Clara Motschenbacher and her family received frightening news. The teenager had Hodgkin’s lymphoma, a rare cancer that affects the lymphatic system. But according to Valley News…
A team headed by Harvard Medical School scientists has taken steps to unravel a dementia that surfaces early in a person’s life. The study, published in Neuroscience News and Nature…
Currently, there is no known cause for multiple sclerosis (MS). While many doctors consider MS to be an autoimmune disease, nobody knows what prompts this immune response. A combination…
It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation…
Before you read on, make sure to check out Part 1 of our interview where we discuss what SLC6A1 is and Aubrey's diagnostic journey. Today, we'll be discussing what it's like to…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, we discussed Dr. Sumeray's background, what hypoparathyroidism is, and its symptoms and treatments. Today, we're…
Both Libtayo (cemiplimab) and Keytruda (pembrolizumab) have been shown to be beneficial treatment options for patients with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). But could Opdivo (nivolumab)…
When Erika Vandenberg learned that her daughter Aubrey had a rare genetic disorder called SLC6A1, her first two reactions were fear and relief. Relief because they finally had a name…