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,…
Spinal muscular atrophy (SMA) is a genetic condition which affects between 10,000 and 25,000 in the United States. While rare, it is currently the leading genetic cause of infant mortality. But,…
Nonalcoholic fatty liver disease (NAFLD) is a condition which causes fat to accumulate in the liver. Nonalcoholic steatohepatitis (NASH) is a severe form of the disease in which the patient also…
Canavan disease is a type of leukodystrophy frequently found in the Ashkenazi Jewish population. It's a rare genetic condition which is ultimately fatal. It's all caused by a deficiency of the aspartoacylase…
Emily Sadler is only 15. In March of 2017 she was diagnosed with thyroid cancer. This came as a huge shock to herself and loved ones, especially because thyroid cancer…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Happy Thursday! We hope everyone's week is going well. Today, we're highlighting a contribution from a cystinosis patient on the necessity of third-party medical providers. We're also sharing a story…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
Being diagnosed with any serious condition can be scary, overwhelming, and full of unknowns. For those who are diagnosed with a rare disease such as Duchenne muscular dystrophy, spinal muscular atrophy,…
Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
While medical students may go into the profession dwelling on the lives they can save, the inevitable fact of the profession is that there will be times when they…
Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed a significantly improved platelet count response after treatment with Cablivi® according to the New England Journal of Medicine's Phase 3 trial results. A…
According to an article recently published by Forbes, one data scientist wants to explain why data science has proven to be slow at identifying particular biomarkers in medical patients. Imran…
As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…
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