Growth hormone deficiency (GHD) influences patients both physically and mentally. The condition means patients' bodies don't produce enough growth hormone. As a result, they don't develop the same way as…
A study published in the Journal of Clinical Oncology has detailed the benefits of different forms of treatment for pediatric patients with adrenocortical carcinoma. These researchers particularly looked at what treatments…
Ryan Alam was diagnosed with mitochondrial-membrane protein-associated neurodegeneration (MPAN), a rare form of neurodegeneration with brain iron accumulation (NBIA), but he hasn't let these conditions slow him down. He has just…
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…
Medical research is crucial to expanding an understanding of certain conditions, which ultimately leads to improved diagnostic and treatment options, and more social awareness. In a recent press release, CurePSP…
How effective is guselkumab, a human monoclonal antibody, at treating patients with psoriatic arthritis (PsA)? According to post-hoc data analysis from two clinical trials, the therapy was relatively effective.…
According to a story from thehansindia.com, 47 year old Xaverine Mukabaranga of Rwanda, Africa, checked herself into Manipal Hospitals. She reported symptoms of persistent chest pain affected her left side.…
According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company…
The Rare Disease Legislative Advocates (RDLA) held its monthly webinar on April 22, 2021. In this informational webinar, the organization featured several speakers who provided updates on some of the…
Peripartum cardiomyopathy is a rare heart ailment, characterized by a weakened heart muscle, that occurs during pregnancy. But why do some pregnant women develop this condition while others do not?…
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…
Over the last 50 years, doctors have been working to improve clinical outcomes for patients with a rare but aggressive form of ocular cancer: uveal melanoma. However, there has been…
In a recent press release, biotechnology and gene therapy company LEXEO Therapeutics ("LEXEO") shared that its gene therapy LX1001 received Fast Track designation from the FDA. Overall, LX1001 is…
If you hear lithium, what's the first thing you think of? For years, lithium has been one of the most widely used and studied therapies for patients with bipolar…
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…
In a news release from March 2021, international pharmaceutical company the Menarini Group shared that it had joined a partnership with Nippon Shinyaku Co., Ltd. ("Nippon") to develop and commercialize…
Happy Friday! Did you know Patient Worthy has a podcast? In the latest episode, we discuss rare disease dermatology with Dr. Steven Chen, MD of Harvard Medical School. Pemphigus and…
According to a story from insidesources.com, many in the chronic fatigue syndrome/myalgic encephalomyelitis community has been very grateful for the news that the National Institutes of Health (NIH) has committed…
Solna Braude describes her brother’s struggle with neurological disorders in her interview with Cure PSP. Her brother, Laurence, was no stranger to the medical world. He was a surgeon, cornea…
According to the Pharmacy Times, patients with chronic inflammatory demyelinating polyneuropathy (CIDP) now have a new and potentially more effective treatment option. After submitting its supplemental Biologics License Application (sBLA),…
William Frackrell’s desire to serve his country was born out of his years as a boy scout and his military career. Then, according to a FOX 13 exclusive with William’s…
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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