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…
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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 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…
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 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…
According to a story from BioSpace, CTI BioPharma Corp. has recently concluded an End of Phase 2A meeting with representatives from the US Food and Drug Administration (FDA). This meeting…
The Sleeping Bear Dunes National Lakeshore Park, located in Michigan, has launched an innovated program to make their park more accessible to those living with a disability. They call it…
According to a story from BioPortfolio, the biopharmaceutical company CStone Pharmaceuticals has recently announced that China's National Medical Product Administration (NMPA) has issued approval for the company to conduct a…
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Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
Last week, the New England Journal of Medicine published a report about a rare, disease-like infection that is a real cautionary tale. A 41-year-old British woman was diagnosed with acanthamoeba keratitis…
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shrouded…
Some promising news from across the pond! A new international consortium funded by the European Union will focus on speeding up the diagnosis and development of rare diseases and treatments.…
Learning that you or a loved one has a rare genetic disease can be scary. Doctors often are not very well versed in these types of disorders and may require…
Throughout my daughter Kelley’s 45-year life with a rare disease, there were numerous times that we needed to either call 911 or take her to the Emergency Department at a…
15-year-old George Taylor reached out to NY Giants Running Back Saquon Barkley, telling the star NFL player how he's an inspiration to him for when he undergoes his chemotherapy for Ewing's sarcoma,…
According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…
According to a story from Science Daily, a recent study has found that osteosarcoma, a form of bone cancer, displays similar genetic characteristics when it appears in both humans and…
The Dog Days Are Back Is it hot or what!? Whether you're trying to catch a tan or stay cool, we have a fresh dose of weekly content in Editor's…
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia…
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