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…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Ahead of Cystic Fibrosis Week in the UK, 37-year-old Marc Cotterill, who lives with the disease, has given a video presentation to the European Cystic Fibrosis Conference on the theme…
Mobility issues are common in patients with osteogenesis imperfecta (OI) - especially tendinopathy, or tendonitis. But why does this happen? According to Medical XPress, a study performed by Baylor College…
For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…
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…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Although it is still considered an experimental technique, gene therapy shows promise for treating a wide variety of genetic diseases or disorders. In a recent news release from the Sanford…
Over the past few years, Elmiron (pentosan polysulfate sodium, "PPS"), a therapy designed to treat patients with interstitial cystitis (IC), has been associated with potential vision damage. Despite a variety…
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…
We still don't fully understand a number of rare diseases. A lack of awareness, lack of interest, and lack of funding all play a role, and medical professionals simply can't…
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…
From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was held fully online. However, that…
Some conditions or diseases are more prevalent in certain groups. In the case of idiopathic intracranial hypertension (IIH), new research suggests that it is much more common in Black or…
Normally, cerebrospinal fluid (CSF) surrounds our brain and spinal cord, providing numerous benefits from injury protection and nutrient delivery to waste removal. Hydrocephalus, or "water on the brain," occurs when…
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…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Evoke Pharma focuses on gastrointestinal disease treatments. Eversana helps to provide commercial services for the life science industry. The two companies have just joined together to launch a social media…
In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…
Could a specialized cancer vaccine actually improve patient outcomes? According to a news release from biotechnology company AIVITA Biomedical, Inc. ("AIVITA"), the answer could be "yes." Citing data from a…
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…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Typically, idiopathic intracranial hypertension (IIH) is associated with obesity, or a body mass index (BMI) over 30.0. As obesity rates have continued to increase globally, so have the incidences of…
Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company…
At just four years old, Gabriel Spiro is facing his 3rd open-heart surgery. You see, Gabriel was born with hypoplastic left heart syndrome (HLHS), a rare and severe congenital heart…
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