Hypoparathyroidism Hypoparathyroidism (HP) is caused by low levels of the parathyroid hormone (PTH). Most people develop this disease after thyroid surgery as a result of accidental damage/removal of the parathyroid…
According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression of x-linked adrenoleukodystrophy in women…
Content Warning: The following article discusses sexual abuse. According to a story from Buzzfeed News, many former patients of an esteemed medical researcher named Reginald Archibald, who was affiliated with…
In order to begin clinical trials for an experimental new drug, pharmaceutical companies in the U.S. are required to submit an Investigational New Drug (IND) application to the FDA. They…
According to a story from IT Business Net, the medical company KIYATEC, Inc., has recently announced that that the Knight Cancer Institute at Oregon Health & Science University has joined…
Tuberous Sclerosis Tuberous sclerosis complex (TSC) is a rare condition which results in the buildup of tumors in the skin, kidneys, brain, as well as other organs. Although the tumors are…
Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…
The Importance of Speech The ability to speak gives individuals not only the opportunity to communicate with one another, it provides an outlet for expression, creativity, and connectedness. Unfortunately, that…
Merkel Cell Carcinoma Merkel cell carcinoma is a rare and aggressive form of skin cancer. It's most often caused by the Merkel cell polyomavirus. However, it can also occur as…
According to a story from pm360online.com, The National Brain Tumor Society (NBTS) recently awarded $750,000 to the Global Coalition for Adaptive Research. This cash is slated to go towards the…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a…
According to a story from MDedge, a review of patient data found that patients with osteomyelitis were more likely to need amputation within a two year period if they had…
Emmanuel Mignot, who is the director of the Stanford Center for Sleep Sciences and Medicine has long believed that some onsets of Narcolepsy could be caused by a confused autoimmune response in…
Narcolepsy and Cataplexy Narcolepsy is a neurological condition that results in a malfunctioning sleep-wake cycle. It affects approximately 185,000 individuals in the United States alone. Some of the most common…
Pheochromocytoma and Paraganglioma Pheochromocytoma refers to a type of tumor that arises from chromaffin cells. They normally occur in the adrenal medulla. However, 10% of these tumors occur outside of this…
Hypereosinophilic syndrome Hypereosinophilic syndrome (HES) refers to a group of rare conditions characterized by high levels of eosinophils which result in organ damage. These eosinophils often affect the lungs, heart, skin,…
About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
ANCA Vasculitis News recently reported findings published in the journal Arthritis and Reumatology on the study “Efficacy and Safety of Belimumab and Azathioprine for Maintenance of Remission in ANCA‐Associated Vasculitis: A…
According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
According to a story from The ME Association, a recent debate that came to the floor of the UK parliament highlights the misinformation and deficits in care surrounding children with…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
deCODE Genetics, which is a research subsidiary of Amgen genomics based in Iceland, has recently created an extremely detailed genetic map. They wanted to uncover more about the human genetic…
According to a story from Renal & Urology News, a recent study found that monitoring of albuminuria, a condition in which the protein albumin appears in the urine, can help…
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…
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