Retinitis Pigmentosa Retinitis Pigmentosa is a rare condition which progressively causes vision loss. Genetic lesions which first affect the rod photoreceptors first cause vision loss at night. Next, cone photoreceptors are…
Gene therapy is a recent development in the industry, and it is one that has brought hope to many people who live with a genetic condition without a viable treatment…
VISEN Pharmaceuticals has recently announced that enrollment has been completed for their Phase 3 clinical trial examining the safety and efficacy of TransCon hGH compared to daily rhGH in patients…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In a case study published on dovepress.com, a 17 year old girl diagnosed with COVID-19 developed Guillain-Barre syndrome, a neurological disease causing muscle weakness, as her symptoms progressed. While COVID-19…
A study published in Physician's Weekly posed the question: is removing milk from the diets of children with eosinophilic esophagitis a preferable treatment option? A team of researchers, led by…
According to BioSpace, a consulting firm called Fact.MR has conducted a study into the market for acute hepatic porphyria (APH). The point of this study was to understand the future…
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A decade ago, Rob Long's dreams were centered solely on football. He was looking forward to the NFL draft after a successful college career at Syracuse University, but unfortunately, a…
A recent study has found that thrombosis may be the first manifestation of granulomatosis with polyangiitis disease in children following the very first report of its presentation in a 13…
A recent study has found that aerobic and strength training exercise programs can have a long term effect on physical functioning for patients with pulmonary hypertension (PH). PH is a…
FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track…
According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…
According to Medical Xpress, a team of researchers from The Wenner-Gren Institute at Stockholm University and Karolinska Institutet, Karolinska University Hospital has conducted a study that investigates neuronal death in…
Prime editing, a new entry in gene-editing technology, is said to be an extension of the “genetic toolbox.” According to a recent article in Science Daily, prime editing designs…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Researchers have been able to identify similarities in outcomes between colorectal liver metastases that cannot be completely removed by surgery (unresectable) and patients with the KRAS mutation. Surgical resection…
According to Healio, an open-label study of oral octreotide has reported that the treatment is able to maintain its response in acromegaly patients without additional safety signals. This data is…
C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
Lyme disease is a vector-borne disease spread through ticks. It is most common in the Northeast, North-Central, and mid-Atlantic portions of the country, but is notably absent in the Southeast.…
Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
A recent study published in Frontiers in Immunology investigates the immune response to bone infections, such as Staphylococcus aureus, that cause osteomyelitis. Their research stands to give medical professionals a better…
A study published in Physician's Weekly investigated the visual outcomes, clinical features, and treatment of individuals with occlusive retinal vasculitis (ORV), a condition that is characterized by inflammation in the vessels of…
During clinical trials, Independent Data Safety Monitoring Boards (DSMBs) sometimes assess trial safety, trial integrity, and how well the trials are upholding patient needs. According to Business Insider, the DSMB…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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