As originally reported in Mayo Clinic, Dr. Veronique Belzil realized she wanted to leave her job as a psychologist to find a cure for amyotrophic lateral sclerosis when she saw…
The Patient Seminar Series is an event in which those who are affected by rare diseases in some way share their experiences. Patients, family members, medical professionals, and legislators all…
Happy Valentine's Day! We hope everyone's enjoying some treats and appreciating the people they love today-- whether that's a romantic partner, a friend, or a family member. We're starting out…
According to a story from mmm-online.com, a recent infographic highlights the challenges that rare disease patients worldwide still face when it comes to getting the treatment that they so desperately…
NeuroSense Therapeutics, a biotechnology company, is in the process of developing PrimeC. PrimeC was created for the treatment of amyotrophic lateral sclerosis (ALS), and it is meant to slow or…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
Having a rare disease means you could have one of any of 5,000-7,000 distinct disorders, each with their own quirks. Just how many there are though remains unclear. As originally…
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Scientists in four continents worked for over a decade on a massive project that has resulted in the most detailed picture of cancer ever produced. Researchers now have a huge…
The National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) of the Food and Drug…
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Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients may be able to receive…
According to a story from curetoday.com, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal: finding better treatments for cholangiocarcinoma,…
As originally reported in StatNews, Mark Zuckerberg, founder of Facebook, along with his wife, Dr. Priscilla Chan, a former pediatrician and now head of the Chan Zuckerberg Initiative, made an…
According to a story from Financial Buzz, the biopharmaceutical company miRagen Therapeutics has recently announced the release of new data from its phase I clinical study. This trial is testing…
As originally reported in ArcaMax, when you get a diagnosis for a terminal rare disease, a wave of emotions crashes over life. Brian Wallach emerged clutching 'hope.' In 2017, Brian,…
Phase 3 Trial A Phase 3 trial run by Pharnext examining the safety and the efficacy of an investigative therapy called PXT3003 began in December of 2015. It enrolled 323…
According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…
Asfotase alfa is an enzyme replacement therapy meant to directly address the cause of hypophosphatasia. While more research is necessary, it has been shown to decrease the mortality rate and…
Happy Thursday! This week, we're highlighting a story about a story about Karina, an EDS patient and advocate. Next, we have a story about a legal controversy that could affect…
According to a story from USA Today, 61 year old Jerry Devries is currently the oldest living patient with a very rare disease: Proteus syndrome. An unusual disease with only…
According to a story from Pulmonary Advisor, the results of a recent analysis led researchers to conclude that systemic anticoagulation therapy can be beneficial for patients with idiopathic pulmonary arterial…
Hereditary Angioedema Hereditary angioedema (HAE) is a rare disease which causes swelling episodes throughout the body, including the airways, gastrointestinal system, and extremities. Most commonly, HAE is caused by a…
According to a story from Angioedema News, a team of researchers and scientists have successfully developed a tool that is able to measure disease activity in hereditary angioedema that is…
According to a story from newswiretoday.com, the biopharmaceutical group Ipsen has recently announced its decision to halt its dosing of patients in its phase 3 clinical trial and its phase…
As originally reported in BioSpectrum; the World Health Organization (WHO) has just approved the disease code for CDKL5 Deficiency Disorder (CDD), adding it to the list used worldwide for the…
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