A Phase 3 clinical trial run by Pharnext for Charcot-Marie-Tooth disease Type 1A (CMT1A) has just been awarded 13.3 million dollars. $7.2 million comes from existing shareholders and $6 million comes…
Rare disease therapies have an exorbitant cost. Fewer patients means higher costs in order to support the development of treatments. In the United States, 966 billion was spent in 2019…
124 million cases worldwide, with 2.73 million associated deaths. These are the numbers for COVID-19, a novel coronavirus pandemic which, over the past 12-15 months, has swept the globe. In 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…
During the Society of Gynecological Oncology 2021 Virtual Annual Meeting on Women’s Cancer, researchers presented data from the Phase 2 OVARIO clinical trial. According to Cancer Network, the trial evaluated…
Chronic kidney disease (CKD) affects hundreds of millions of people throughout the world. As of 2017, 697.5 million people were estimated to have CKD by this study published in Nature Reviews…
COVID-19 has caused long-term, ongoing symptoms for some patients. Up until recently, these individuals had been called "covid long haulers." Anthony Fauci, during a White House briefing, has recently changed…
An estimated 13-20 out of every 100,000 people around the globe have idiopathic pulmonary fibrosis (IPF). The condition often has a poor prognosis (3-5 years following diagnosis) and there are…
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Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
First of all, a little about me. I’m an Australian, living in the Netherlands with my German husband and our 6-year-old, identical twin daughters. We’re a pretty average family, living…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
As the pandemic shifted cocktail hour onto zoom and professors posted their full course material online, many rare patients reveled. Not because we weren’t suffering through the pandemic too, but…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
Rare Disease Day is February 28th. This year, it caused a lot of reflection among healthcare professionals and families impacted by rare conditions. How has the pandemic shifted progress in…
In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…
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 late March 2021, the Hypersomnia Foundation and biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") partnered up to launch the "I Have IH" campaign. The campaign is designed to raise awareness…
bluebird bio has created a gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD), which is now making its way through a clinical development program. According to Businesswire, bluebird has…
A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…
The drug development process is not always easy or quick. It consists of drug discovery, preclinical and clinical research, and FDA approval. However, sometimes developers are sent back to the…
How much should medication cost, and are these treatments cost-effective and accessible for patients? In a press release, biopharmaceutical company Aurinia Pharmaceuticals, Inc. ("Aurinia") shared that an independent review performed…
Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…
The majority of Parkinson’s patients treated with Levodopa experience relief from the major motor effects of Parkinson’s. However, according to a recent article in Biospace, after prolonged use of Levodopa…
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