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…
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…
In 2001, I developed severe chronic pain from a neurological movement disorder called dystonia. My life before and after dystonia are as different as night and day. I went from a very active lifestyle to…
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.…
According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…
Eltrombopag (Promacta) has now been approved for all individuals in the US with severe aplastic anemia. The drug will be accessible to both pediatric and adult patients. This is huge news.…
According to a story from CNBC, 53-year-old James Addie has been living with hemophilia since the day he was born. Because hemophilia is a genetic disease, the blood disorder is…
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…
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