According to a story from finanznachrichten.de, the UK drug development company AstraZeneca recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for the company's medical…
According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…
The following story includes discussion of suicide. According to a story from EurekAlert!, a recent study has revealed that survivors of head and neck cancer are four times more likely…
According to an article published by CheckOrphan, LEO Pharma and PellePharm have announced a new multi-million dollar partnership together with the aim of advancing research for rare skin diseases that…
The Asia-Pacific Economic Cooperation (APEC) has 21 members including the United States, China, Canada, Australia, Peru, Russia, and Thailand to name a few. They work to encourage the interdependence of the…
The world is recognizing more every day just how important rare disease research is. Yes, rare diseases are rare but they affect millions of people worldwide. The more we can…
According to a story from tgen.org, a team of scientists from the Translational Genomics Research Institute (TGEN) have recently initiated a study that will test the effectiveness of liquid biopsy…
According to a story from Health Canal, a team of researchers led by Dr. Mohammad Siddiqui of Virginia Commonwealth University (VCU) recently released a study which further highlights the relationship…
Last week the Biotechnology Innovation Organization released a letter to the FDA, commenting on the latter's draft of a document that proposes a new set of guidelines for the treatment…
According to a story by reporter Nara Schoenberg from the Chicago Tribune, when Pidgeon Pagonis, then known as Jennifer Pagonis, was born in 1986, it didn't take long for Pidgeon's…
According to a story from the Farmington Daily Times, Dr. C. Frank Bennett, who grew up in Aztec, NM, and his collaborator Dr. Adrian Krainer, were both jointly awarded the…
You might have heard of the recent political situation where President Donald Trump criticized retired Adm. William H. McRaven on a number of issues. We're not going to dig into…
According to a story from Business Wire, the drug developer Cycle Pharmaceuticals recently announced that is has completed its goal to obtain the necessary data regarding the stability of its…
While most of us have trouble accepting our limitations, it can be especially hard for people whose limitations are due to a rare disease. In meeting with and corresponding with…
"Do you know what the world’s third most populated country is?" It's a question that Dr. Rafael Sousa Fernandes posed in an OpEd, wrote that made all of us here at Patient…
According to a story from BioSpace, the biopharmaceutical company Sobi and the biotechnology company NovImmune SA have recently announced that the US Food and Drug Administration (FDA) has approved Gamifant,…
According to a story from the biotechnology company Amgen, a review from the European Medicines Agency (EMA) has recommended that the indication for blinatumomab, marketed as BLINCYTO®, be expanded. The…
According to the US Centers for Disease Control and Prevention, there have been 10 more confirmed cases of acute flaccid myelitis (AFM), a polio-like illness that mostly affects children. This now…
The Newborn Screening Saves Lives Act was originally introduced to facilitate the spreading of awareness and education related to newborn disease screenings. The screening of newborns has had a major…
According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…
We are entering the Thanksgiving season. I’ve been taught all my life that it shouldn’t be only a season, but an attitude year around. But how? By telling Mom thank…
According to a story from CheckOrphan, a wide array of experts are raising concerns about India's rare disease policy, which was first unveiled in May of 2017. Despite being hailed…
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
According to a story from tonic.vice.com, Liz Aronin and Jamie Mills both thought that everything was going smoothly with their newborn son Quinn, but within a few months, it became…
99% Invisible recently reported on the history of "Orphan Drugs." These drugs are an enormous source of income for pharmaceutical companies - but the explosion in their production is a…
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