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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During the time of a pandemic, it is valuable to look to past pandemics and judge their effects. Eleven years ago, H1N1 swept the world just as COVID-19 is now.…
Lyme disease is typically cured with antibiotics, and those affected never experience symptoms again. But for 20% of those who contract this illness, the antibiotics are ineffective, resulting in lingering…
According to a story from BioSpace, GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical, Inc. have recently announced that the first patient has been dosed in the companies' phase 1/2 clinical trial.…
According to a story from Yahoo News, Dr. Nancy Wexler spent around 20 years in Venezuela conducting pivotal research on a deadly rare genetic disorder: Huntington's disease. On Lake Maracaibo,…
My American Nurse recently carried an uplifting article showing increasingly positive changes for cystic fibrosis (CF) patients. A network of over one hundred thirty centers throughout the U.S. provide…
Continued from Part 1 The surgery was over 12 hours long. I have two rods and 26 screws holding my spine and had nine ribs cut and reduced in…
A recent editorial focusing on NMDs (neuromuscular disorders) was published in the journal Disease Models & Mechanisms. The editorial highlights a special issue of the journal devoted to building…
According to a story from LocalHealthGuide, COVID-19 has continued to spread through the US and the lackluster government response suggests that it isn't going to be contained any time soon.…
I was born in 1974 in Buenos Aires, Argentina to Italian immigrants. I was born with a rare genetic disorder called Ehlers-Danlos syndrome kyphoscoliosis type, but it was not diagnosed…
Antonio Maltese is a young man living in Virginia. His family has also been devastated by the hereditary rare illness called Huntington's disease. In fact, he was diagnosed with the…
As originally reported in Biospace, there’s good news today for adult patients with acute hepatic porphyria in Europe, a genetic disorder which causes pain and paralysis. The European Commission has…
According to a story from The Charlotte Observer, Chris Younger was 18 when he first was diagnosed with X-linked hypophosphatemia, a rare disease that affects the bones. However, he had…
"When women come together to demand change, change happens." These are Dr. Svati Shah's words at the Miss America 2020 pageant. She encouraged the watching audience that although cardiovascular…
By Danielle Bradshaw from In the Cloud Copy. Many of us probably don’t think too hard about our medical records on a day to day basis; which is fair -…
By Danielle Bradshaw from In the Cloud Copy. Right before Thanksgiving last year, Sami Winters, an East Bay resident, was admitted to Benioff Children’s Hospital in Oakland, California. It’s now…
By Natalie Homan from In The Cloud Copy Mara Clawson is a visual artist who uses many different mediums to create art pieces that are full of color and life.…
By Jodee Redmond from In The Cloud Copy The results of a case study have found that dantrolene can be used to treat involuntary muscle contractions (dystonia) in children with…
LymeDisease.org is one of the most distinguished nonprofits for Lyme disease in the United States. In 2015 they created MyLymeData in the effort to improve treatments for this disease. 10-20%…
According to a story from Yahoo Finance, the biopharmaceutical company Mallinckrodt recently released findings from an open label pilot study which tested the impact of Acthar Gel (corticotropin injection) as…
According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the…
According to a story from Healing Hugs Haven, the rapid spread of COVID-19 around the world has been getting a lot of airtime in the news lately, and for good…
AVROBIO has recently received the Orphan Drug designation for AVR-RD-04, a gene therapy for the treatment of cystinosis. This treatment takes the stem cells of those affected and modifies them…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
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