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 Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…
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…
According to a publication from The Rheumatologist, the recent failure of a seemingly-promising lupus treatment in a phase 2 study has led to a discipline-wide discussion about new research and…
According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…
SMARD is such a rare disease that only 100 children in the entire world have been diagnosed with it. SMARD stands for spinal muscular atrophy with respiratory distress. SMARD…
According to a publication from Fierce Biotech, researchers from Boston Children's Hospital have created a new gene therapy technique they believe could provide benefits to patients with inherited forms of…
Patient Worthy had the privilege of attending another United Leukodystrophy Conference. The ULF’s 2019 event took place at the end of June in Illinois and had a record setting number…
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Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
According to a story from EurekAlert!, in recent years immunotherapy, which are methods of treatment that enhance or harness the body's immune system to fight disease, have become increasingly prominent…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc has announced the release of preliminary topline data findings from its phase 2a clinical trial, which tested…
In a time where we get bombarded by all sorts of troubling news -- it's so soul-refreshing to see these truly inspirational moments go viral. A video of limb different,…
In an exclusive "Good Morning America" interview this week, Dr. Priscilla Chan announced the launch of Rare As One, a new program from the Chan Zuckerberg Initiative where she is co-CEO…
According to a story from Pulmonary Arterial Hypertension News, data from an open label extension trial of the drug Orenitram as a treatment for pulmonary arterial hypertension has demonstrated that…
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The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…
TGIF! Unless you're on vacation in which case, every day is *basically* Friday. This week, we're bringing four articles to read as you escape the summer heat. We have a…
According to a story from Verdict Medical Devices, CVS Health has recently initiated a clinical trial which will test a device called HemoCare. This device is being developed by DEKA…
According to a story from Fabry Disease News, a recent study has found that despite changes to sperm cells, the fertility of men with Fabry disease is not affected. The…
The Immunization Action Coalition (IAC) Express Newsletter reports that 981 cases of measles have been reported so far this year, with 41 new cases added just last week. Rare disease kids…
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