Pharnext SA, a biopharmaceutical company, recently announced results in a PLOS ONE article entitled "Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A…
According to a publication from Stat, scientists have high hopes about "virtual repurposing" cutting drug production costs. It could also potentially mean more treatments on the market faster. Virtual Repurposing…
According to a report from ITV, a five-year-old boy from southern England has made the trip across the Bay of Biscay to Barcelona, Spain, where he is receiving an experimental…
Vogmask is a San Francisco, California based company founded in 2011. They create stylish facial masks to protect wearers from inhalation of particulate and allergens. These types of masks can…
This is week three of this season of The Bachelor on ABC, and week three of a patient-related subject highlighted on the show (Colton's CF foundation, Caelynn's childhood encephalitis)-- so I'm…
Read the first part of Lisa's story here. Lisa’s two direct and indirect bypass surgeries were successful. She now had seven times more blood flow to her brain than she…
UPI Health News recently reported that a study, published in the New England Journal of Medicine, showed that fecal microbiota transplantation resulted in a 32 percent remission rate in patients…
According to a story from dailymail.co.uk, the story of Cayla Jones is an example of how a serious rare diagnosis can suddenly strike out of nowhere, often from symptoms that…
Bontulinum Neurotoxins (BoNTs) are a protein that are used to treat a wide array of conditions including cervical dystonia, blepharospasm, neurogenic detrusor overactivity, and sialorrhea to name a few. These proteins…
According to an article published by R&D, scientists at Harvard have identified a new genetic clue that they suspect might be linked to the development of amyotrophic lateral sclerosis (ALS).…
The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
According to an article from Global Banking & Financing Review, the California-based pharmaceutical company Exelixis, Inc. has announced the FDA's approval of their drug Cabometyx (cabozantinib) for patients previously diagnosed…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
According to a story from Biotecnika, Penn Medicine & Inovio Pharmaceuticals Inc. have recently announced that the US Food and Drug Administration (FDA) has signaled its approval for the first…
A new study published in the journal Cell Reports identifies possible new treatments for absence (silent) seizures in children with Dravet syndrome. Children with Dravet syndrome have developmental delays, cognitive…
Mucolipidosis Type IV Mucolipidosis Type IV (ML-IV) is a rare disease which causes a reduced mental capacity and blindness. ML-IV is caused by a missing or malfunctioning TRPMLI protein. Most patients never…
According to a story from dailymail.co.uk, 23 year old Ruby Hill of New Zealand was recently released from the hospital, but it wasn't because she had been successfully treated. It…
According to a story from kios.org, firefighters across the country tend to get cancer, including many rare cancers like mesothelioma, at significantly greater rates than the general population. This is…
According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
According to a story from bioengineer.org, it has long been known that people with the rare disorder Duchenne muscular dystrophy are at a greater risk of developing a rare cancer…
Cameron Dixon is a 17-year-old boy living with muscular dystrophy. His family was first told that he would most likely never be able to walk, eat, or talk, and that…
A couple weeks ago, we wrote an article about the role of Colton's Cystic Fibrosis foundation in this season of The Bachelor. Since my terrible TV taste is now revealed…
What is the Disparity? America is greatly divided by many things and race has always been one of them. Not only are there large social and economic divides among racial…
The University Hospitals Birmingham in the UK is one of the four centres in the country to run a clinic for patients with Bardet-Biedl syndrome. They’ve recently written an article…
Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…
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