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…
A recent article in ANCA Vasculitis News highlighted a Chinese study of ANCA-associated vasculitis (AAV), a group of autoimmune diseases. An earlier Chinese study suggested that the risk of…
A recent study has found that undergoing chemotherapy after having surgery for a rare kidney cancer reduces the chance of cancer relapse by 50%. The recent study was published…
"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…
By Rachel Whetstone from In The Cloud Copy Although people sometimes want to hide away when a disease leaves visible marks on their bodies, Academy Award winning actress Tatum O’Neal…
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%…
Hey, hand-washing friends! We hope everyone is staying safe and clean right now. Of course, the world is reckoning with Covid-19 right now-- and the rare space is no except.…
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…
Gina Hollenbeck never considered that she could have lung cancer. She did not fall into any categories that would put her at a higher risk of cancer; she didn't smoke,…
Roche has recently been granted priority review by the FDA, who has also accepted their supplemental biologics license application for atezolizumab. Atezolizumab is intended for the treatment of non-small cell…
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 businesswire.com, the Bristol Myers Squibb Company recently released the topline results from its phase 3 clinical trial. This study was testing a combination treatment consisting…
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…
As originally reported in Targeted Oncology, a man was diagnosed with EGFR-Mutated Non-Small Cell Lung Cancer, also known as EGFR-NSCLC. The 60 year old had a previous medical history that…
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…
The world is coming to terms with climate change and the vast number of changes that will come as the earth slowly turns up the heat. A change of this…
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…
According to a story from Financial Buzz, the biopharmaceutical company Mereo BioPharma Group plc recently announced that it has successfully concluded its Type B End-of-Phase 2 meeting with the US…
In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…
By Danielle Bradshaw from In The Cloud Copy Pompe disease is the lack of a specific protein within the body that helps break down glycogen - a complex sugar that…
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