According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in medical theory, but only in…
According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene therapy treatment that was approved…
According to a story from The Intercept, Gilead Sciences' antiviral drug remdesivir, which is being tested as a treatment for coronavirus/COVID-19, was just given Orphan Drug designation by the US…
As originally reported in European Pharmaceutical Manufacturer Magazine, Chiesi, an Italian pharmaceutical company, has announced they will be launching a new division for rare disease research and therapy innovation. The…
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…
According to a story from Healio, a recent study that was published in the scientific journal BMC Rheumatology revealed that it was uncommon for patients living with ankylosing spondylitis to switch from…
As originally reported in Live 99.9; when Melissa and Chris Courson found out the baby they were expecting in a few months would be born with the rare thantophoric dysplasia,…
By Jodee Redmond from In The Cloud Copy Massachusetts Institute of Technology (MIT) researchers have recently announced they have identified hundreds of genes that are required for neuron survival. The…
People affected by rare diseases often find it difficult to find adequate treatment for their conditions. Due to the rarity of their diseases, there are often no doctors who specialize…
As originally reported in Edinburgh Evening News; Assed Ramae felt the depths of familial love when he received his younger sisters kidney, saving him from the decline from his stage…
By Rachel Whetstone from In The Cloud Copy New research is exploring the relationship between circadian rhythms in the brain and bacterial fragments. It’s possible that bacteria has an influence…
By Natalie Homan from In The Cloud Copy Spina bifida is a condition that appears when a developing baby’s neural tube doesn’t close completely. The most severe form of spina…
It's easy to feel helpless right now. You're told to stay home and just wait it out. So you're stuck feeling isolated and angsty, knowing there's nothing you can personally…
Chronic granulomatous disease (CGD) is a rare form of immunodeficiency. The most common form of this disease is X-linked chronic granulomatous disease (X-CGD) which is caused by a mutated CYBB gene. This…
The Fighting HARD Foundation Rare diseases are difficult to face for a myriad of reasons. They can be financially burdensome, physically painful, and emotionally draining. Further, they can make patients…
The European Parliament has announced that they will be relaunching their "Network of Parliamentary Advocates for Rare Diseases." Its aim is to create a new policy framework which can improve…
Researchers all over the world are hard at work trying to develop both treatments for Covid-19 and vaccines for the illness. Professors of rheumatology at the University of Alabama, Randy…
There are currently no FDA approved treatments for alopecia areata, a condition that sees the loss of hair all over the body. Eli Lilly and Company, working with Incyte Corporation,…
We know that older individuals and those who are immune compromised are more at risk for experiencing severe Covid-19. These include multiple sclerosis (MS) patients. Multiple Sclerosis is a rare…
Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…
By Danielle Bradshaw from In The Cloud Copy There are not too many things that a mother won’t do for her child, and Alison Reynolds is the perfect example of…
Pyoderma gangraenosum (PG) is a rare auto inflammatory disease which affects the skin. It unfortunately has no currently approved therapies. However, recent preliminary results from a Phase 2a trial have…
The first patient has been dosed in the third phase of the VIITAL study, which is intended to evaluate the effectiveness of EB-101. This medication is a gene-corrected cell therapy…
The Problem with Identifying the Genes that Cause a Genetic Disorder from In The Cloud Copy When it comes to genetic disorders, it can be very difficult to pinpoint exactly what…
To celebrate Rare Disease Day and raise awareness for approximately seven thousand rare diseases, Dr. Francis Collins, director of the National Institutes of Health, recently interviewed a person he…
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