According to a publication from Medscape, the first ever drug targeted for bladder cancer has shown even more impressive results in post-approval study. Erdafitinib was approved on an accelerated basis…
We love a good story about a community coming together! Firefighters across the Atlanta area came together to support a retired fire chief who has been partly paralyzed after being…
According to a story from Infectious Disease Advisor, a study has found that infection with the hepatitis C virus and cryoglobulinemia (high levels of cold sensitive antibodies) can lead to…
According to a story from Charcot-Marie-Tooth Disease News, Charcot-Marie-Tooth disease is actually somewhat common in the US compared to other rare diseases. In fact, it is estimated that around 150,000…
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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 they can rely…
August 3rd is CLOVES Syndrome Awareness Day! The CLOVES Syndrome Community (CSC) is an organization that "supports, educates, empowers and improves the lives of those affected by CLOVES Syndrome" and…
According to a press release from British biotechnology company Immunocore, the Company has partnered with Pulse Infoframe (a Canadian data analytics company) to support the development of an international database…
According to a story from Cancer Health, a team of scientists affiliated with Mayo Clinic, UC San Francisco, and the Chan Zuckerburg Biohub have discovered a new rare, autoimmune disease…
Happy Thursday! This week, we're highlighting a PW contributor's story exploring blepharospasm and mental health and an article on two sisters with a rare connection. Following that, we have a…
I had the pleasure of attending the Glut1 Deficiency Foundation’s 2019 Conference. While this article can't possibly do the event justice and encompass all it had to offer, we will…
The struggle of living with a rare disease and dealing with insurance issues was in the spotlight on national television. During Tuesday night's 2020 Democratic debate for the president, Senator…
According to a story from Angioedema News, a recent clinical trial has demonstrated that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of acute…
According to a story from the Broad Institute, there are still many mysteries to uncover related to inflammatory bowel diseases, which includes the rare form ulcerative colitis. The understanding of…
A Colorado mom is on a mission to raise money for her daughter's type 1 spinal muscular atrophy (SMA) treatment -- that would save her child’s life. Let's hear little Maisie…
According to a story from Angioedema News, a team of researchers has used data from a recent study to provide up to date recommendations for diagnosing hereditary angioedema in the…
An article from Comic Sands recently covered the story of a UK mother, Alison Beesley, who is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
Talking about mental illness is never easy but I wanted to share my experience of it with you. In my case, it came as part of my struggle with Benign…
According to a story from globenewswire.com, the biopharmaceutical company Mustang Bio, Inc. has recently announced that the US Food and Drug Administration (FDA) has recently awarded the company Orphan Drug…
According to a story from drugs.com, the pharmaceutical company Pfizer, Inc. recently issued an announcement detailing the approval of its drug Ruxience (rituximab-pvvr), a biosimilar to the widely used monoclonal…
According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…
According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…
According to a story from rediff.com, Dr. Rahul Desikan was a committed amyotrophic lateral sclerosis researcher. Then, in a cruel twist of irony, he was diagnosed with the disease himself.…
According to a story from Raconteur, as many as 3.5 million people living in the UK are expected to affected by some type of rare disease at some point during…
According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…
According to a publication from Charcot-Marie-Tooth News, the first patient has been enrolled in the first ever natural history study of type 4J Charcot-Marie-Tooth disease. Study collaborators — biotechnology company…
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