According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…
According to a story from arkansasonline.com, Rep. Julie Mayberry R-Hensley of the Arkansas General Assembly is sponsoring House Bill 1074. This legislation proposes adding the rare and potentially fatal genetic…
According to a publication from Breastcancer.org, black women are more likely than others to develop triple-negative breast cancer at some point in their lives. A study published by the journal…
Progress Report Debuts ASCO’s Research Priorities for the Cancer Community, Emphasizes Impact of Federal Funding FOR IMMEDIATE RELEASE January 31, 2019 Ashley Yum 571-483-1376 [email protected] ALEXANDRIA, Va. — Over the…
We've all seen the commercials - especially during the holiday season - for home DNA kits like 23andMe and Ancestry DNA, where a saliva sample can unlock your family heritage.…
Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
According to a story from The New York Times, a new wave of research is starting to reveal the close connection between the human gut microbiome and the brain. Some…
Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…
February 2nd is Neuroblastoma Awareness Day! It's important to know more about this rare cancer, since knowledge and familiarity are often the engines of progress. What is Neuroblastoma? Neuroblastoma is a…
According to a publication from PR Newswire, CloudMedx, a technology company from Palo Alto, is touting impressive results following a simulated exam pitting human doctors against their "deep learning" Clinical…
There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal. These diseases include phenylketonuria, homocystinuria, tyrosinemia, and maple syrup…
Rare disease families deal with a lot. The last thing they should be forced to have on their mind is how to get access to care. Unfortunately, for many parents…
A recent survey conducted by researchers at the NYU School of Medicine has indicated that oncologists know much less about the unique cancer needs of the LGBTQ population than they thought.…
According to a story from falmouthpacket.co.uk, couple Geoff and Judie Davis were devastated when their two year old boy Mikey died after being diagnosed with neuroblastoma. That was more than…
According to a story from BBC, it is becoming increasingly clear that a lot of the common phrasing and language that we use to talk about cancer and cancer patients…
In a story published in The Hernando Sun, Dawn Dybiec, age 31, describes her disbelief when she and her husband were told that her two sons, Gabriel and Steven, were…
Mesothelioma is a cancer which occurs in the thin layers of tissue (mesothelium) which surround most of the body's internal organs. It's often caused by asbestos exposure. However, symptoms can take…
Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process…
Ionis Pharmaceuticals and Roche have been collaborating on a drug for Huntington's Disease which will treat the root cause of the condition. Currently, there are no such approved treatments, meaning…
While juvenile idiopathic arthritis remains to be the most common rheumatic disease in children, researchers have long been stumped at its caused. The best prediction was that it originated as…
According to a story from 2 Minute Medicine, a recent analysis found that patients with lupus nephritis saw their risk of death decrease by 70 percent on average after receiving…
Non-tuberculous mycobacterial (NTM) infections are caused by bacteria found in the environment. In healthy individuals, these bacteria normally cause no harm. However, for those who have a compromised immune system or who…
Happy Wednesday! While it's felt like January was going to last forever, we're almost through the first month of 2019. As we gear up for February, we want to reflect…
A rarely addressed subject that comes up in discussions about people with disabilities is the subject of romantic relationships, intimacy, and parenting. More than likely, a lot of day to…
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