According to a story from FOX31 Denver, Kelly Conley of Arvada, Colorado saw her circumstances change dramatically when she first began experiencing symptoms of Cogan's syndrome, a rare disease that…
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 the Hindustan Times, seventeen year old Yash Galpelli was born with Wilson's disease, a rare genetic disorder. For a long time, he had no idea…
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…
According to a story from wect.com, local singer Dustin Chapman of Whiteville, NC has been struggling for years against a rare disease called achalasia, which affects the esophagus. He recently…
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…
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare disease that affects the nervous system. It causes peripheral nerves and nerve roots (the sites where nerves are attached to the central…
Ehlers-Danlos Syndrome Ehlers-Danlos syndrome (EDS) refers to a group of rare disorders which affect the connective tissues in the blood vessels, bones, skin, and other organs in the body. It's caused…
A Brazilian company known as Dr. Consulta is taking healthcare to a place only previously known by fast food chains. The company, founded in 2011, breaks the mold as a…
Tristan Lederman's health started to take a turn at the beginning of 2017. Tristan lived at home with his family and depended on his family members and at-home caregivers…
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,…
One of my better friends within the cystinosis community is Mika Covington. We met at a cystinosis event in 2016. I had read some of Mika’s entries posted in her…
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…
A recent article in CheckOrphan reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the synthetic GLP-2 analog apraglutide for the treatment of short bowel syndrome…
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…
Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…
We’ve all become a little more in tune to what’s good for our bodies, and what isn’t. Part of that is medical science finally being able to identify our bodily…
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…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
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…
According to a press release from X-Rx Inc., a privately-held American biotechnology company, the Food and Drug Administration has accepted the company's Investigational New Drug (IND) application for X-165, an…
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