According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This has naturally…
A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
According to a story from offspring.lifehacker.com, medical abnormalities and conditions that affect the skin are often something that many of us will notice on our own. Once a skin problem…
Rare Chronic Illness Support Group 2nd Friday of each month This is a peer support group for patients living with chronic, rare diseases. Featuring Carrie Pope, MSW, LICSW To sign…
Geisinger Health System is based out of Pennsylvania. They have recently initiated a novel program that has goals to make DNA sequencing a standard part of routine clinical care. Next…
According to a story from birminghammail.co.uk, eight month old Archie was diagnosed with a serious rare disorder called Pompe disease. With his life in danger, the infant faces 220 mile…
Web-based Rare Chronic Illness Support Group 2nd Thursday of each month This is a peer support group for patients living with chronic, rare diseases. Featuring Cara Oppegard, MSW intern To…
According to a story from derbytelegraph.co.uk, four year old Michal Myslicki, who was born with Batten disease, will soon get access to a critical treatment that will prolong his life.…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This had naturally…
NASH Nonalcoholic Steatohepatitis (NASH) is a rare disease in which hepatic inflammation and injury to the cells occur as a result of steatosis (hepatic fat accumulation). Severe cases of NASH…
By: Teresa Scruton Hereditary Angiodema Abdominal Attacks There are 3 types of Hereditary Angiodema (HAE) abdominal attacks: Lower abdominal - swelling in wall of colon (large bowel), that causes the…
Web-based Living Mindfully With Chronic Illness Support Group 2nd Tuesday of each month This is a support group for rare disease patients dealing with chronic illness who are seeking to…
According to a story from Wales Online, Samantha Josey's childhood was characterized by unpleasant symptoms and health problems, resulting in hospital visits and bouts of serious illness. Her symptoms included…
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Waukesha Wisconsin Police Captain Dan Baumann recently held a news conference that was described in an article published in Scientific American. Captain Baumann said that parents of a teenage boy showed…
The Center for Chronic Illness is a nonprofit group that is based out of Seattle, Washington. The goals of this organization include the provision of support and educational resources to…
With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
Cystinosis Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body's organs. However, there is treatment that can prevent this accumulation. Normally, patients…
Ischemic cardiomyopathy is a type of dilated cardiomyopathy, in which the capability of heart to pump blood is reduced. This causes lack of blood supply to heart muscles which result…
In a story from Healio, Dr. Niraj C. Patel, who is an associate professor of pediatric infectious disease and immunology at Levine Children’s Hospital, Atrium Health in Charlotte, NC, is…
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FierceBiotech recently reported that Helixmith Co., based in Korea and previously known as ViroMed, coordinated a Phase 3 clinical trial involving five hundred diabetic peripheral neuropathy (DPN) patients. The…
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