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…
Primary biliary cholangitis (PBC) is a rare disease, but you don’t have to face it alone. Join us for an educational program and connect with others who understand what you’re going…
Researchers at Columbia University have found a way to apply CRISPR, a tool used in gene therapies, to new types of disease, reports News Medical. The study was carried out…
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
A California family has lost 12 family members to the newly discovered rare disease known as hereditary sensory and autonomic neuropathy type 1E (HSAN 1E), which involves a mutation on…
Here's something on the lighter side of disease prevention efforts! Earlier this month, the Centers for Disease Control and Prevention tweeted a photo of a poppy seed muffin which was meant…
May is national Cystic Fibrosis Awareness Month! Cystic fibrosis (CF) is a genetic condition that causes progressive respiratory and digestive system damage. Someone with CF has thick, sticky mucus, versus…
According to a story from Science Daily, a recent study found that a rigorous exercise program reduced fatigue in testicular cancer patients and helped boost their self-esteem. Patients with the…
According to a story from Medscape, the House of Representatives recently voted to pass a "right to try" bill. The bill they voted on was previously approved by the Senate…
The Alagille Syndrome Alliance (ALGSA) is preparing to host its first annual gala, The Gala of Dreams, this year on Saturday, June 30th, 2018 at the San Diego Marriott Del…
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