To read part one of this story on cystinosis, click here. Briana and Ashley are two adult women from opposite regions in the United States. Briana lives close to her…
According to a story from Reuters, the Danish biotech company Genmab saw its share value drop by a quarter after Johnson & Johnson, which had partnered with the company to…
She wears a lacy white dress with a long, white veil. Her beautifully shaped eyebrows arc over long-lashed eyes. Her smile, illuminated by bright red lipstick, speaks of the happiness…
A new study conducted by Centers for Disease Control and Prevention found that premature babies were more susceptible to having obstructive sleep apnea (OSA), reported RadioAllen. About 1 in 10 newborns…
Researchers have carried out a study into the genetic mechanisms involved in hereditary angioedema acute attacks. They found that two genes, called uPAR and ADM, had a particularly strong influence.…
Happy June everybody! We hope everyone's ready to kick off their summer, whatever that may include. This week we have two stories from younger patients who are leveraging their own…
According to a story from apnews.com, President Trump signed the "Right to Try" bill passed by Congress into law on Wednesday, May 30th, 2018. During the signing ceremony, he was…
Research into possible facial creams containing the medicine rapamycin have found that they are effective for reducing facial tumours in people with tuberous sclerosis complex. The full article can be…
According to an article from Globe Newswire, the company Shire plc recently presented new data in relation to recombinant human parathyroid hormone (rhPTH) and its relationship to hypoparathyroidism. This information…
After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…
According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…
According to a story from news-medical.net, Mariona Graupera and Frances Viñals, along with Dr. Antoni Riera-Mestre, announced the completion of research that could lead to a new potential treatment for…
May 15 to June 15 is designated as Tourette Syndrome Awareness Month, a rare neurological disorder that causes involuntary movements or vocalizations known as tics. Examples of these tics include repetitive blinking, clearing…
The United States Food and Drug Administration (FDA) has granted the drug tafamidis Breakthrough Therapy designation for treating patients who have transthyretin cardiomyopathy. The full article can be read here,…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the Phase 2 trial for its proprietary, investigational product sotatercept. The medication is in development…
The PBCers Biannual Conference recently took place in Houston, Texas. The 2018 PBCers Conference was highly informative with topics ranging from the basics of Primary Biliary Cholangitis, dietary tips, updates…
Fresh off of Memorial Day, there is no better documentary to watch right now than HBO's John McCain: For Whom the Bell Tolls. The Arizona senator announced last July that he…
When others learn that you are the parent of a child with a rare disease, they most likely correctly assume that doctor appointments and hospitals might be involved. Yet few…
I'm Celeste, and I'm sharing my story because too many ill people go undiagnosed and too many healthy people know nothing of it. Being chronically ill is like (for lack…
According to a story from Ovarian Cancer News, a recent study has allowed scientists to determine why some people appear to be resistant to poly ADP ribose polymerase (PARP) inhibitors.…
The not-for-profit healthcare organisation Sanford Health is planning to implement routine genetic testing for patients to identify a patient’s risk for a range of rare, and more common, diseases. The…
The Young family from Oregon, U.S. have used genetic screening to test their embryos for a variation of the BRCA gene that significantly increases the risk of breast cancer, reports…
TGIF, everybody! This week we're sharing music videos by a singer who raises awareness for aHUS, a journey of a dysautonomia patient, and a story about a man who was…
May has been designated Behçet's disease awareness month - but unlike so many of the rare diseases we cover, there are not any advocacy organizations dedicated to raising awareness and support…
According to a story from the Washington Post, an outbreak of Nipah virus has claimed a total of ten lives so far in south India. Medical teams are starting to…
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