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.…
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…
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…
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…
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…
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…
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…
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…
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…
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…
According to a story from The Oncology Times, the US Food and Drug Administration (FDA) has been moving forward with several treatments and potential treatments for a variety of different…
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…
According to a story from MarketWatch, Annie Wilson was diagnosed with spinal muscular atrophy when she was only six months old. With no real treatment options available, doctors told her…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a story from prnewswire.com, the drug developer Mirum Pharmaceuticals recently announced that is has entered an agreement with Shire which grants exclusive rights for marketing and development of…
According to a story from ncbnewyork.com, a recent study found that $7 million on donations on crowdfunding sites for medical patients have gone towards treatments that are medically unsound and…
According to a story from Xconomy, the Rainwater Foundation, which was first founded in the 90s by Texas billionaire Richard Rainwater, is offering cash rewards to the tune of $250,000…
According to a story from Buzzfeed News, Paige Rogers was 21 years old when she was admitted to the hospital. She was having trouble breathing because of cystic fibrosis. Over…
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
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