July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
According to a recent press release, biotechnology company Cancer Prevention Pharmaceuticals (CPP) submit a New Drug Application (NDA) for accelerated approval of their drug therapy CPP-1X/sul to treat familial adenomatous…
Without treatment, spinal muscular atrophy (SMA) is the top cause of infant or child mortality before age 2. As a result, early detection is crucial in improving patient outcomes. But…
This morning, SynAct Pharma AB announced in a press release that they began a Phase II clinical trial to determine the efficacy and safety of AP1189 on treating patients with…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently approved a new treatment for Dravet syndrome, a rare disorder that causes seizures and…
It amazes me how quickly the numbers change each time I write about COVID-19, the novel coronavirus declared a global pandemic in March 2020. As of the afternoon of…
According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…
Last week, I had the privilege of attending a webinar organized by Research!America titled Partnership, Progress, Pandemic: The Impact of COVID-19 on Medical Discovery. As implied in the title, the…
According to a story from Biotech 365, the biopharmaceutical company Deciphera Pharmaceuticals has recently announced that the company's drug ripretinib (marketed as QINLOCK™) has been approved by Health Canada. The…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has just approved a treatment for rare adult-onset Still's disease for the first time ever. The…
According to a story from Buzzfeed News, the results of a recent trial held in the UK may have finally revealed a medication that can have a major positive impact…
In the geographically small island nation of New Zealand, there are estimated to be as many as 300,000 people who live with various rare diseases or disorders. Unfortunately, like in…
According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month. While physical distancing as a result of the ongoing coronavirus/COVID-19 pandemic may keep some folks at…
An estimated 25-30 million Americans are part of the rare disease community. If you're one of them, you share a common (yet uncommon) experience: public scrutiny, difficulties in diagnoses, treatment…
Education, research, support, and patient advocacy: these are just four of the amazing things that rare disease or disorder foundations offer. Many foundations seek to offer programs and resources to…
Last night, I texted my mom to ask what blood type I was. "Type A, I think? I'll have to check. Why?" she texted back. Well, I had just read…
As much as you and I have tried to keep up on COVID-19 news, the information changes every day. Although about 80% of cases are asymptomatic or mild to…
In a press release from Eli Lilly and Company, the company has announced recently that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (BLA)…
June marks the start of Aphasia Awareness Month, designed to educate many on aphasia: causes, long-term affects, and the lives of those living with this condition. But, as the University…
Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
May 16th is celebrated annually as Hereditary Angioedema (HAE) Awareness Day. The event was established with the goal of spreading awareness about this rare condition among both the general public…
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