In a world that is just beginning to make space for the open discussion of transgender individual's life experiences, there's still a huge gap in the conversation regarding healthcare. This…
There was a time when most children diagnosed with a rare condition did not live until adulthood. But now, with advances in medicine, many individuals are living long, full lives…
According to an article from Stat News, patients, doctors, and advocates around the country are starting to push more energetically for a repeal of a U.S. law that effectively bans…
Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…
According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the…
The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
Happy Thursday! We hope everyone's spring is off to a good start! As the weather warms up, we're sharing stories demonstrating patient hope, strength, and resilience. We start with a…
In 2012 Tessa noticed a gap between her front teeth. She was a little confused. She had had braces when she was younger and her teeth hadn’t had any gaps…
Anna Landre is a sophomore at Georgetown University in Washington, D.C. It’s a good school, you’ve probably heard of it. On top of the normal stresses of her college life,…
Neurodegenerative Diseases There are a wide range of neurodegenerative diseases including Huntington's disease and Parkinson's disease. These conditions typically affect the older population. As the world's population ages, more people…
According to a story from EurekAlert!, effective management of Parkinson's disease can be a serious challenge. Part of the reason that the management of the illness is so difficult is…
Background Novartis ended all of their gene therapy programs which were oncology related in 2016. Unfortunately, that meant an end to many programs that still had potential. This included cell…
According to a press release from British biopharmaceutical manufacturer Autolus Therapeutics, the American Food and Drug Administration (FDA) has granted the Company's experimental acute lymphoblastic leukemia (ALL) cell-based immunotherapy treatment…
According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The…
According to a report from ArsTechnica, scientists believe that some people might have medicinal poop. What might sound like a bad punchline to a child’s joke is actually the result…
According to a story from Top Class Actions, the drug companies Biogen, Inc. and Abbvie recently released a recall for their multiple sclerosis drug Zinbryta following several reported cases of…
Results from the largest United States study to date of Lambert-Eaton myasthenic syndrome (LEMS) have indicated that for the majority of patients, Firdapse is the most effective treatment option currently…
Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…
According to a story from EurekAlert!, a group of researchers affiliated with Penn Medicine and Children's Hospital of Philadelphia recently conducted an experiment in which the scientists used CRISPR gene…
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Although guidelines for Parkinson’s patients suggest that they visit a specialist every six months, according to a report in Parkinson’s News Today, the waiting period may be up to…
According to a story from inpublic.globenewswire.com, the Novartis company AveXis has released interim data from an ongoing Phase 3 clinical trial of Zolgensma, a gene therapy drug that is being…
Lyme disease is an infection by a type of bacteria called a spirochete. This type of bacteria is carried by deer ticks and when an infected tick bites an…
According to a story from globenewswire.com, the biopharmaceutical company TG Therapeutics, Inc. recently made an announcement declaring that the US Food and Drug Administration (FDA) had awarded the company Orphan…
When you’re diagnosed with a rare condition it can change the entire course of your life, at least at the beginning. But those living with diseases like these are fighters.…
According to a publication from KYN24, the Food and Drug Administration recently approved mucopolysaccharidosis type VII (MPS VII) drug Mepsevii for marketing and administration to patients in the United States.…
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