According to an article from the JAMA Network, the role of the human microbiome in our health is just beginning to be understood, and the manipulation of the human gut…
According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…
Happy Thursday! We're back with a batch of new article! This week, we have another article from PW contributor, Tom Seaman, who uses his experience with dystonia to help people…
AI Therapeutics is a biopharmaceutical company that has created an artificial intelligence platform that is meant to match drugs to new indications. This platform, called Guardian Angel, has matched…
According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…
Gene therapy has become a technological golden snitch: the end of an era for many genetic disorders if we can grasp the treatment’s potential. However, the technology comes with a…
According to a story from News Amed, Rob Burrow gained fame as a star player for the Leeds Rhinos, a professional rugby team. After a distinguished career, Rob retired from…
January 24th is Moebius Syndrome Awareness Day! Let's take a deeper dive into what Moebius Syndrome is and what we can do to push awareness on this day (and all…
One of our partners, the MDS Foundation, Inc, is sponsoring another free MDS Patient & Family/Caregiver Forum. This event will be held in Birmingham, Alabama When: Saturday, February 1, 2020…
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According to a recent article in Yahoo News, Johan’s mother’s seven-year-old brother died years ago of chronic granulomatous disease (CGD). The mother, Maren, was told that she also had…
As originally reported in Quest Qunam and Sure Scripts; for manydiseases, time is of the essence in terms of treatment. The patient's health will deteriorate irreversibly thout treatment. However, with…
ALS is a progressive disease that causes the loss of muscle function, and it often develops very quickly after a diagnosis. Rami Epstein, the CEO of the biopharmaceutical company called…
Schizophrenia patients in the United States now have a new treatment option. Intra-Cellular Therapies has just announced that their investigative therapy called CAPLYTA has received FDA approval for adult schizophrenia…
According to a story from outsourcing-pharma.com, the US Food and Drug Administration (FDA) has recently started a new initiative with the opening of its Compounding Quality Center of Excellence. Janet…
According to a story from Medgadget, a team of scientists from Columbia University have been working on a robotic device that could assist patients with spinal cord injuries and impairments…
January is Cervical Health Awareness Month! The New Year holiday is all about taking stock of one's self and making efforts to improve or set goals for a better you.…
Doctor Daniel Kastner, director of NHGRI, recently uncovered a new rare disease along with his research team. It’s called CRIA syndrome. This discovery has been published in Nature. CRIA CRIA…
Happy New Years! We're back with a batch of new article! This week, we're highlighting four stories: an interview on 20 years with dystonia, a young woman stuck in a…
As originally reported in EurekAlert, creating effective rare disease pharmaceuticals is difficult. Yes, because it is expensive, there’s a small population to use in trials, and there’s limited research. Sometimes…
The human spirit can be a remarkable force. Robb Freed cycled thousands of miles by himself to raise awareness for epidermolysis bullosa (EB) -- a rare illness that took the…
The annual Code Talker award was presented during an engaging ceremony at the National Society of Genetic Counselors’ annual conference. Every year, patients submit essays nominating their genetic counselors for…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
As originally reported by the Oklahoma Medical Research Foundation; Tonja Martin was running through her retirement savings for her medical care. She spent $3,400 every month to cover treatment for…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
Who is most affected when we talk about creating solutions for rare diseases? Those who live with them and their families. Patient communities know what they need and feel the…
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