As reported in PR Newswire, Alcyone Therapeutics is launching a new set of precise gene therapies for CNS disorders with the help of a new $23 million investment from RTW…
As seen in PR Newswire, researchers at the biopharmaceutical company OxThera AB have spent 52 weeks studying 25 patients with primary hyperoxaluria (PH), a rare disease that progressively damages the…
Liver cancer has been one of the leading causes of cancer-related deaths. A recent article in HemOnc Today reports that liver cancer is perhaps one of the most difficult cancers…
In a press release by Parkinson's Canada; researchers in Canada wanted to unpack why some people first lose their sense of smell before developing Parkinson’s disease with age. While losing…
Researchers from the University of Lausanne and the Wellcome Sanger Institute have just published a new study in Molecular Systems Biology which uncovered how DNA can overcome genetic changes which are…
Many rare disease patients are familiar with high prices for treatment. In fact, the most expensive medication in the world treats a rare disease: Zolgensa, a gene therapy for spinal…
Komodo Health and PicnicHealth have just announced that they have partnered together in effort to combine their data and better serve patients. This data will then be made accessible to…
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…
According to a post on WAMES (Working for ME in Wales), a recent study used a retrospective analysis to identify potential warning signs for a characteristic symptom of myalgic encephalomyelitis…
Recent research conducted by Dr, Elif Oral has discovered that leptin therapy may be a viable treatment for nonalcoholic steatohepatitis (NASH), which is a form of fatty liver disease. Dr.…
The gut plays a big role in our bodies, we know that. While some of its roles are obvious, others are still being discovered. In fact, the microbiome of the…
According to a story from mdlinx.com, a recent study has found that around 20 percent of sudden cardiac death cases in US adults can be linked to pathogenic arrhythmia syndromes…
According to a story from Parkinson's News Today, an approach called whole-body electromyostimulation (WB-EMS) was able to benefit Parkinson's disease patients in a small scale study. These patients were not…
Pfizer’s Regional President for Rare Disease, Reda Guiha, recently offered his opinion to The Parliament magazine about the urgency of what he refers to as the ‘rare disease crisis.’ Pres.…
A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…
ACADIA Pharmaceuticals has released data on NUPLAZID, a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. This treatment is meant to treat Parkinson's disease patients, specifically the delusions…
According to a story from MedPage Today, a recent study has determined the familial hypercholesterolemia (FH) can often go undetected on genetic tests that rely on arrays. As an example,…
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…
Medical professionals are still trying to understand all of the underlying causes of Parkinson's disease, a progressive disease that sees the degeneration and death of motor neurons. A recent study…
Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…
Sobi as well as the Hellenic Institute for the Study of Sepsis have just announced successful results from their SAVE-MORE study for those with COVID-19. The results were from 28…
A recent study in MedicalXpress investigates a mutation that contributes to Parkinson’s-like symptoms in children. The mutation interrupts the movement of dopamine. The new study was conducted using fruit flies…
BeiGene has just announced that China has given conditional approval to Pamiparib, a PARP inhibitor used as a therapy for BRCA associated ovarian cancer, fallopian tube cancer, and primary peritoneal…
Enzyvant has just announced that they have resubmitted their application of RVT-802 to the FDA as a therapy for pediatric congenital athymia. Their first application was submitted in 2019. The…
Diagnosis can be difficult to achieve for rare disease patients. Some patients experience years of searching for the correct diagnosis, with others receiving the wrong one along the way. Changes…
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