VistaGen Therapeutics has recently been cleared by the FDA to begin the second phase of their study of AV-101, which is intended for the treatment of dyskinesia in those affected…
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…
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…
A study published on February 3rd, 2020 in the scientific journal Nature Research is highlighting the capability of a three part combination treatment for use in multiple myeloma, a rare and…
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 a report in Myeloma Research News, the European Commission has recently granted authorization to market bortezomib, dexamethasone and thalidomide (collectively known as VTd) plus daratumumab to treat…
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Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…
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…
According to a story from Malaysian Tribune, the pharmaceutical company Khondrion has just announced that the first patients have been dosed in its phase IIb clinical trial. This trial is…
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…
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…
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Researchers at the Medical Center of Hackensack University conducted a study of three drugs now proven to be safe and effective for the treatment of severe seizures. According to…
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.…
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,…
According to a story from consultant360.com, a recent study was conducted in order to determine if racial ancestry had any impacts on outcomes in the rare disease pulmonary arterial hypertension…
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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According to a recent article in MedPage Today’s 2019 year-end review, the number of life-threatening lung diseases in systemic juvenile idiopathic arthritis (sJIA) patients has been increasing. sJIA is…
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