My name is Megan and I live with acute hepatic porphyria (AHP). My health journey began with the arrival of puberty. I started to experience terrible nausea and abdominal pain…
While novel treatments have been rapidly emerging onto the drug scene, new treatment options for pancreatic cancer have fallen behind. But now, according to OncLive’s interview with Dr. P.A. Philip,…
Parents of children who have received a diagnosis of Hutchinson-Gilford progeria syndrome (HGPS) may be relieved to learn the FDA recently approved Zokinvy capsules that were developed to lower…
As reported in EMJ Reviews; the novel coronavirus has gripped the world— fueling a wave of medical innovation and prioritization. However, the excess of need has made the normal flow…
According to EMJ, findings from an analysis spanning 20 years has found that weight loss surgery used to address obesity also significantly lowers a person's risk of developing pancreatic cancer.…
As reported in EMJ Reviews, the world is on alert, being told to wash their hands and keep distance amidst the COVID-19 pandemic. However, patients with inflammatory bowel disease (IBD)…
25 year old Tylia Flores wrote on her blog that as a person with cerebral palsy, there are a lot of not so thoughtful ‘compliments’ that may not be interpreted…
The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
Immunoglobulin A nephropathy (IgAN) is a glomerular disease which can lead to kidney failure. Between 70 and 100 percent of all IgAN patients also are diagnosed with microscopic hematuria, but…
Super T’s Mast Cell Foundation was created in 2015 by my daughter Taylor upon receiving her diagnosis of mast cell activation disorder. After traveling over twenty hours to consult with…
According to a story from Charcot-Marie-Tooth News, a recent cell-based study has revealed that Charcot-Marie-Tooth disease type 1C and type 4J share a common pathway malfunction. This is despite the…
Hayden Grosvenor is used to spending time in the hospital. He has Langerhans cell histiocytosis (LCH), a rare condition that is characterized by an excess of white blood cells. Because…
Grant Bonebrake was diagnosed with Alport syndrome at age 12. Since then, he has gone on to advocate for not only Alport syndrome patients, but the rare disease community as…
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…
Research During COVID Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due…
Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
The Partnership A new partnership has just been announced between Mendelian, a digital health company for rare disease, and Modality NHS partnership, a rare disease diagnosis program. Together, these organizations…
Neurodegenerative diseases such as Parkinson’s, Alzheimer’s, and multiple sclerosis are disorders that mostly affect the neurons located in the brain. These diseases are incurable, resulting in the death of nerve…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
Toxicity resulting from cancer therapy is usually the result of the repeated administration of cancer drugs. On the other hand, the efficacy of CRISPR-Cas9 has been largely affected by problematic…
According to a recent press release from biopharmaceutical company Praxis Precision Medicines ("Praxis"), two of the company's drug candidates received either Orphan Drug or Rare Pediatric Disease designations for rare…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
A recent announcement by Gilead Company citing data from Kite Pharma, one of its premier companies, set out remarkable results from Kite’s Zuma-1 clinical trial. The findings confirmed that…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
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