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…
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…
There is a high unmet need to find more effective treatments for liver cancer. Currently, treatment options for patients often only increase life expectancy by 3 to 4 months. An accidental…
According to a story from The People's Pharmacy, there are an increasing number of worrying stories and incidents that are calling into question the US Food and Drug Administration (FDA)'s…
Hope everyone's week is going well! Today, we're highlighting an article discussing the difficulties of securing a CIDP diagnosis, followed by an update on a Niemann-Pick Type C treatment. Next,…
According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in…
According to a story from the New York Times, a 50 year old Houston man had seen his health decline rapidly in a little over a year. After declining from…
According to a story from Scientific American, the World Health Organization (WHO) recently decided to strike the diagnosis congenital Lyme disease from its International Classification of Diseases (ICD). The document is…
According to a story from philly.com, it was August 2018 when Lisa Semple of Cherry Hill was first diagnosed with breast cancer. Unfortunately, her breast cancer had reached stage four,…
According to a story from Science Daily, an experimental therapy for cancer could shut down tumor cells using an innovative mechanism that disrupts their circadian rhythm functions. A concept study…
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