According to a story from Southwest Journal, retired professor Karen Warren was first diagnosed in 2015 with multiple system atrophy, a lethal neurodegenerative disorder. The disease carries an unpleasant group…
A recent article written by Craig Klugman debates the ethical obligations pharmaceutical companies have to conduct clinical trials for drugs which have a likely potential to benefit a patient population…
According to a story from kdrv.com, cystinosis is a very rare disease that only affects about 500 people in the US. Two of those patients are Emma Suetta and Everly…
According to a story from curetoday.com, the findings from a recent study indicate the patients with metastatic bladder cancer demonstrated an improved rate of response to an immunotherapy drug combination…
According to a publication from EurekAlert, a recent study helmed by the Royal Marsden NHS Foundation Trust suggests that female cancer patients tend to live slightly longer following treatment than…
What are Generic Drugs? Generic drugs are any medications "equivalent" to brand name pharmaceuticals. These drugs are much cheaper than the brand name medications and the United States Food and…
According to a story from blog.cincinnatichildrens.org, a diagnosis of Duchenne muscular dystrophy is always an alarming piece of news; learning that your child has a progressive muscle atrophy disorder that…
According to a publication from Yahoo News, five doctors interviewed by the media organization have reported meeting with a number of migrants detained by United States Customs and Border Protection…
The Importance of Braille For any child with a rare disease that leads to blindness or visual impairment such as Stargardt disease, Usher Syndrome, Alström syndrome, or Retinal Blindness life…
According to a story from Healio, data from two studies testing autologous tumor infiltrating lymphocytes (TIL) as a treatment for advanced, metastatic cervical cancer and melanoma suggests that this treatment…
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
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…
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