According to a story from The British Journal of Cardiology, the use of renin-angiotensin-aldosterone-system (RAAS) inhibitors or blockers is a valuable component of treatment for many medical conditions and diseases,…
Pretoria Moot Rekord recently reported on the 35th anniversary of the University of Pretoria's Facial Cleft Deformity Clinic. The clinic caters to babies and children with facial clefts, providing surgery…
In 2013 researchers discovered something interesting about the protein BH4. They found that by inhibiting the protein they could reduce patient's pain. A drug was created called Quartet, and it…
According to a publication from MarketScreener, pharmaceutical titan Pfizer Inc. has gained FDA approval for Daurismo, a once-a-day oral medication designed to be paired with low-dose cytarabine (a type of…
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…
According to a story from Business Wire, a recent survey has revealed new findings about the frequency of neurogenic orthostatic hypotension (nOH) among Parkinson's disease patients. Around one in five…
According to an article originally published at The Rheumatologist, a CRESPA (that's Clinical REmission in peripheral SPondyloArthritis) study recently showed that golimumab, a TNF inhibitor, might be effective in relieving…
According to a story from MedicalXpress, it was around 30 years ago when two sons of Marsha and Allen Barnett, named Chuckie and Michael, both passed away from Leigh syndrome.…
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…
4D110 has just received Orphan Drug Designation for the treatment of Choroideremia (CHM). The phase 1 trial of the drug is expected to start in 2019. What is Chorioderemia? Chorioderemia is…
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 the Des Moines Register, Iowa has a new medical marijuana program rolling out on December 1st, but many doctors in the state have reservations about enrolling patients. Iowa's…
According to a story from uhb.nhs.uk, a recent study has drawn a link between chronic kidney disease and Alström syndrome. The research team is associated with the University Hospitals Birmingham…
Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…
According to a story from BioSpace, medical student Kristin Hunt of McGill University will be this year's recipient of the David R. Cox Scholarship for Rare Compassion. The purpose of…
A frequent impediment to rare disease research is cost. It's unfortunate, but it's the reality of the world we live in. That means every fundraising campaign and every dollar raised…
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