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…
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 globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is…
According to a story from BioSpace, the biopharmaceutical company Insmed Inc. has recently announced favorable results from its phase II clinical trial. The clinical trial is testing the company's investigational…
According to a story from Chronicle Live, 36 year old Donna Campbell was found unresponsive in her hospital bed on June 22nd, 2019. She was officially pronounced dead the following…
According to a story from globenewswire.com, the biotechnology company Emerald Health Pharmaceuticals Inc., has announced recently that its experimental drug EHP-102 has earned Orphan Drug designation from the European Medicines…
Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…
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…
Lisa Christopher-Stine is the Director of the Myositis Center at Johns Hopkins. She recently shared her biggest tips for patient care and some updates from the field, hoping to change…
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According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…
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According to a January 2020 release by Business Wire, the USFDA has awarded accelerated approval of Epizyme’s TAZVERIK™ in the treatment of advanced epithelioid sarcoma (ES). The approval was…
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 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…
Ipsen is a pharmaceutical company based out of Paris. Sadly, they have just announced that they have pressed pause on not one but two studies for fibrodysplasia ossificans progressiva (FOP). FOP…
The State of PTO Of all of the industrialized countries in the world, the United States is the only one that in the year 2020 still doesn't guarantee that workers…
Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have…
Khondrion has just announced that the very first patient has been dosed in their Phase 2b study for mitochondrial diseases called KHENERGYZE. This patient population has a high unmet need…
The Benefits of Genomics The use of genomics in health systems is becoming more of a standard practice. Health systems have been partnering with genomics databases to collect patient data.…
As originally reported in NewsRoom, the US's political volatility over the past years has thrown many policies and legislation into debate. While switching the party in both the executive and…
FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They've successfully provided millions toward research initiatives and clinical…
International Alagille Awareness Day The very first International Alagille Awareness day was held on January 24th, 2020. What is Alagille syndrome? It is a rare disease which effects the heart,…
India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…
Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…
The Platform Notable's automated technology platform was created to help predict which patients would respond better to which therapies. As no patient is the same, this study is paramount. Notable…
According to a story from Federal Telemedicine News, there are around 7,000 diseases known to science that can be considered a rare disease. When we consider these diseases in the…
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