In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107…
The FDA advisory panel which is composed of specialists who advise the FDA on brain medications, recently voted 6-0 in agreement on that the new drug, Leqembi, was effective…
According to a story from PR Newswire, the QurAlis Corporation, a clinical stage biotech company, recently announced the completion of the review of the Clinical Trial Regulation (CTR) in the…
Sophie Hinchcliffe, best known by her online moniker of Mrs. Hinch, knows how to clean up a mess. After all, her cleaning skills are what launched her into popularity…
In 2019, Adam Sorgi began teaching psychology, health sciences, and AP psychology at Capistrano Valley High School: the same school that he had attended as a teenager. That same…
Right now, there are insufficient treatment options for familial chylomicronemia syndrome (FCS). Unfortunately, this means that individuals living with this rare genetic disease are left without much assistance. The…
Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. The…
Earlier this year, I interviewed Dr. Arthur Suckow, the CEO and co-founder of DTx Pharma, about the innovative RNA therapeutics being developed for Charcot-Marie-Tooth disease type 1A (CMT1A). In…
The Progeria Research Foundation reports that as of March 2023, there are 193 children and young adults living with progeria or progeroid laminopathies in 51 countries across the globe.…
Relyvrio was officially approved by the FDA for treating ALS in September 2022. However, according to an article in BioPharma Dive, there was considerable controversy surrounding the clearance of the…
An announcement by Pfizer was recently published in Biospace outlining the results of BASIS its Phase 3 clinical trial (NCT03938792). The drug being studied, marstacimab, met its primary endpoints…
Céline Dion’s new milestone is her first-ever film, Love Again, which she stars in while living with a rare disease called stiff person syndrome. Céline expressed her wishes that…
810 words (source) vs. 506 words (mine) - 2% match Chemotherapy, radiation, and surgery may all be utilized to help patients to manage nasopharyngeal carcinoma (NPC), a rare and aggressive…
According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
According to a story from CBS News Boston, Scarlett is just six years old and was recently hit with a devastating diagnosis: Batten disease. Unfortunately, very little can be done…
A court in Zabbar, Malta found the parents of seven-year-old Victoria guilty of her death by negligence. The court also determined that the system had failed the child by…
Ticks can carry a number of disease-causing pathogens. If an infected tick bites a human, that human can then contract several illnesses. In many cases, people who contract these…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a Phase 1 study. Within this…
Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB subtypes with their own inheritance…
Thirty-year-old Brendan O’Reilly was told that his rare disease affects only one person in 250,000. He was also told that he is lucky to be alive. Brendan’s symptoms confused…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem manganese is largely being developed…
On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…
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