Many faced with a rare disease or condition often experience financial burdens in seeking out adequate educational tools, therapeutic devices, medical equipment, resources, treatment options, and more. As described by…
A child's life was recently saved thanks to ultra-ultra-rapid genome sequencing which provided him his rare disease diagnosis in just 13 hours. The Human Genome Project began 30 years ago…
Yeast can be used in baking, brewing, and fermenting. But could it also be used to treat patients with inflammatory bowel disease (IBD)? According to Medical XPress, doctors believe so.…
Jo Blaney was diagnosed with cyclic vomiting syndrome when she was pregnant with baby number four. She is now striving to bring awareness to the condition, which impacts patients' daily…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
During the European Hematology Association (EHA)'s 26th Annual Meeting, taking place virtually, biopharmaceutical company Kite (part of Gilead) shared longer-term follow-up data on Yescarta (axicabtagene ciloleucel) for patients with relapsed…
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…
Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…
CRISPR is a gene-editing technology which holds the potential to treat a variety of genetic diseases and improve patient outcomes. Overall, CRISPR works by creating precise cuts and edits within…
In a news release from June 29, 2021, biotechnology company Neurogene Inc. shared that its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) received Orphan Drug designation from…
According to the Centers for Disease Control, one in ten people are carriers of a disease-causing bacteria called Neisseria meningitidis. In most cases, they will not experience adverse effects. However,…
An exciting article in the Guardian published last year and a confirming article published this month state that new diagnostic technology has shown ‘impressive results’. This test can spot tumors…
People are rushing to get in shape for summer; it's common to see the gyms a little more full than normal. While exercise is good for you, it's important not…
An Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare diseases or conditions, affecting less than 200,000 Americans. Because it can sometimes be difficult…
In the past, antipsychotics were recommended as a potential treatment for patients with Parkinson's disease (PD). This is because, in later stages of PD, patients may experience Parkinson's psychosis, which…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
From June 4-8, 2021, the American Society of Clinical Oncology (ASCO) held its Annual Meeting to discuss trends and research within the oncology field. According to Cancer Therapy Advisor,…
As seen in a press release by Bioindustry; the UK BioIndustry Association (BIA) has published findings from a survey on the public’s interest in supporting rare access to affordable medicine…
Medical professionals have been searching for a viable treatment for Parkinson's disease for a long time. While there have been advancements and new therapies created, many patients are still looking…
Eosinophilic asthma is a rare and often severe form of asthma which can be difficult to treat. Because of this, many patients with eosinophilic asthma experience diminished quality of life…
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…
Federal officials from the CDC and FDA have recently come out to address the possible connection between mRNA vaccinations for COVID-19 and cardiac issues. This recent statement acknowledged that there…
Medpage Today ran the entertaining story of the fabled crow who quenched his thirst by tossing stones in a pitcher of water to raise the water level. Prior to…
On June 22, 2021, PharmaTimes Online reported that the European Commission (EC) granted conditional approval to Koselugo (selumetinib), an orally administered therapy, for use in pediatric patients with neurofibromatosis…
Some good news was reported recently in a Biospace article. Dana-Farber Cancer Institute and Harbour BioMed have agreed to combine resources and co-develop novel biotherapies for oncology and immunological…
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