According to a story from PR Newswire, the biopharmaceutical company Harmony Biosciences, LLC recently announced that the US Food and Drug Administration (FDA) has approved the company's drug pitolisant (marketed…
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 Angioedema News, the drug development company Regenxbio has announced that it is in the process of developing a new gene therapy treatment for hereditary angioedema,…
According to a story from BioPortfolio, the biopharmaceutical company Emalex Biosciences, Inc., has recently announced that the very first patient has been signed up for the company's phase 2b clinical…
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…
According to a recent article in CheckOrphan, glioblastoma (GBM) is considered to be among the deadliest cancers in the world. Currently, treatment options are surgery, chemotherapy, and radiation. In most cases,…
According to a story from Pharma Times, the drug company Ipsen has recently made available a pre-filled syringe of its drug lanreotide (marketed as Somatuline) which will allow patients to…
According to a story from statnews.com, new, groundbreaking forms of treatment, such as gene therapy and CAR-T cell therapy, are giving patients with rare genetic disorders and advanced cancers more…
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…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) has recently announced an exciting new partnership that will help facilitate spreading awareness about Waldenstrom's macroglobulinemia (WM), a rare form of blood cancer. This…
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 Newswise, Quest Diagnostics, a laboratory that specializes in diagnostic information, has announced plans to participate in a unique, large scale trial called NCI-MATCH, which stands…
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 BioSpace, the biopharmaceutical company Immunic, Inc. has announced that the first patient has signed up for its proof-of-concept clinical study. This study is intended to…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
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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…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is actually a term used to classify a few different conditions. Two of these are Crohn's disease and ulcerative colitis. These are rare…
Juvenile idiopathic arthritis (JIA) is a rare condition affecting children. Like all patients, these children are told what treatment they need and then they are expected to comply. However, researchers in…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…
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