Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…
According to a story from Charcot-Marie-Tooth News, a recent study has revealed that patients with Charcot-Marie-Tooth disease (CMT) type 1A still display signs of muscle weakness even if they retain…
According to a story from Medgadget, a team of researchers associated with Yale University have discovered that the symptoms of Tourette's syndrome can be controlled through the use of functional…
TGIF! Something we believe is that the stories of rare disease patients are vitally important. This week, we're highlighting two articles written by patients, as well as a story about…
According to a story from news-medical.net, a recent study is giving scientists new insights into the nature of Merkel cell carcinoma, a rare cancer that affects the skin. The study…
According to a story from Market Watch, the biotechnology company Vertex Pharmaceuticals, Inc. has announced recently that the US Food and Drug Administration (FDA) has accepted the company's New Drug…
According to a story from Comic Sands, mother Alison Beesley is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
Sumaira Foundation Sumaira Ahmed established the Sumaira Foundation so that fewer rare disease patients would have to face the experience by themselves. Sumaira Ahmed is diagnosed with neuromyelitis optica spectrum disorder…
Here's a shocking fact for you- individuals don't always conform to averages. Despite this, we use averages as an analysis for so many things. As it documents the majority, its…
According to a story from The People's Pharmacy, the health dedicated website (along with many other sites that discuss drug effects and side effects) has experienced a nearly 50 percent…
According to a publication from BioSpace, the American Food and Drug Administration (FDA) recently approved the Inrebic (fedratinib) for the treatment of certain myelofibrosis patients. About Myelofibrosis Myelofibrosis is a…
When Amber Freed and her husband Mark resorted to an in-vitro fertilization (IVF) baby they were overjoyed when they found out they were pregnant and were welcoming two new…
According to a story from outsourcing-pharma.com, the company Iqvia has just launched its all-new patient portal. However, while many patient portals are intended to allow for patients to monitor their…
According to a story from The Indian Express, early treatment and diagnosis of primary immunodeficiencies makes management of this group of disease much simpler as a whole. In children, the…
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A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide. The definition of rare disease differs between the United States…
According to a story from kfor.com, Governor Kevin Stitt of Oklahoma recently held a signing ceremony for a bill that is being hailed as a rare example of bipartisan collaboration…
Happy Thursday! This week, we're highlighting an article about the neglect of policies meant to protect people with IBD. Next, we have stories on two recent studies affecting MS and…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…
According to a story from EurekAlert!, the results of a recent clinical trial should get the attention of patients in the US with the rare disorder familial chylomicronemia syndrome. The…
Critical Path Institute The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as…
Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals. The hereditary condition is…
According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…
According to a story from Medpage Today, a recent phase II clinical trial of an experimental dual-action CAR-T cell therapy has yielded an impressive 95 percent response rate. The drug…
At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…
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Trust is vital when it comes to medical care. People put their most important questions and qualms into the hands of doctors and medical staff and expect that they can…
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