Ella Casano is a 12-year-old living with Idiopathic Thrombocytopenic Purpura (ITP) who had a brilliant idea about how she could help other children living with rare diseases like her own.…
Mark your calendars! The Rare Disease Diagnostics Summit is being held in Boston, MA on September 24th and 24th, 2019. This incredible event will bring together pharmaceutical companies, biotechs, diagnostics,…
According to a publication from Medicine Matters, a 12-person task force organized by the European League Against Rheumatism (EULAR) has released a set of 12 recommendations to improve quality of…
According to a story from news-medical.net, an international research team affiliated with the UK Medical Research Council, Indiana University School of Medicine, and the University of Kansas have released a…
According to a publication from Fierce Biotech, Biotech company Sangamo recently updated investors on three trials it has underway testing experimental forms of gene and cell therapies. The trials are…
According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…
According to a story from The Monitor, Nubia Garza's ten year old daughter Edie was diagnosed with the rare condition Williams syndrome when she was just four months old. The…
According to a recent publication from BioPharma Dive, Dr. Janet Woodcock sat for an interview at the Biotechnology Innovation Organization's annual conference in Philadelphia earlier this month. Woodcock, the Director…
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Dr. Craig Klugman, a bioethics professor at DePaul University, expresses his opinion in this recent article in Columbia University’s Bioethics.com. Dr. Klugman begins with a discussion of Pfizer’s biological drug,…
According to a story from Science Daily, a recent study has found that lithium chloride was able to improve muscle size and strength in a mouse mode of a specific…
After years of having multiple physical complications and problems, a young woman in Spirit Lake found medical answers in 2016. In that year, Jessica Brown was diagnosed with Chiari…
For most of us, our body follows a predictable timeline when it comes to growing older. While there's some variation depending on genes and lifestyle, each year, we age with…
According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
Functional neurological disorder is a conversion disorder. This type of complex disorder is often thought to be linked with psychological symptoms, serious stress, or mental distress. With functional neurological…
According to a story from bioengineer.org, a team of researchers associated with Oregon State University are making further headway in understanding the connection between our health and the bacteria that…
Stem Cell Therapies While stem cell therapies hold a great deal of promise for many rare diseases, there is still a lot that we don't understand. Experimental stem cell therapies…
Meredith Resnick's breast cancer risk is 42%. This risk is made up of her higher breast density (denser breasts are associated with increased risk), Ashkenazi Jewish heritage, a family history…
Do you have ADPKD? If you or someone you know has ADPKD, email us at [email protected]. We'd love to hear from you about your experience! According to a story from…
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In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism. Janet Woodcock,…
According to a story from ANCA Vasculitis News, a recent study conducted in Korea determined that ANCA vasculitis patients in the region were at a greater risk of developing thyroid…
"A new way to improve the efficacy of a drug taken by millions of patients throughout the world." Background Adalimumab (also known as Humira) and infliximab (also known as Remicade)…
According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
MVID Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID…
According to a story from Science Nordic, the monoclonal antibody drug rituximab (often marketed at Rituxan) had no effect on patients with chronic fatigue syndrome (CFS). Chronic fatigue syndrome is…
Happy Thursday! Today, we're highlighting four articles on rare disease news. First, we have a story on a lawsuit against controversial stem cell treatments. Next, we have an article on…
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