According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
According to a story from the Translation Genomics Research Institute (TGen), the organization was recently awarded a grant of $10,000 from PayPal Gives. The funding will be allocated towards funding…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…
According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…
According to a story from freepressseries.co.uk, 45-year-old Stephen Millichamp was recently diagnosed with AL amyloidosis. The news was dire, and Stephen was told that the disease would ultimately be fatal.…
According to a story from HudsonAlpha Institute for Biotechnology, a team of researchers affiliated with the organization have identified a genetic mutation that is linked a rare pediatric form of…
According to a story from Financial Review, the US is not the only place in the world where lobbying is a central component of political activity. So far, lawmakers in…
According to a story from EurekAlert!, a collaboration of Sage Bionetworks, The Children's Tumor Foundation (CTF), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) have announced the initiation of the very…
According to a story from EurekAlert!, a team of researchers used a combination of microarray technology and blood tests in order to distinguish distinctive molecular signatures in a group of…
According to a story from Dallas News, Shereese Hickson is a 39-year-old single mother. She has multiple sclerosis, and it seemed like her symptoms were starting to flare up again.…
According to a story from Global Knock, nine year old Ashton Leeds has been living an active life again lately, but a year ago, things were not looking up for…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
According to a story from the National Post, 34-year-old mother Andrea Cardin, a photographer, was excited to welcome her newborn son James into the world on November 21st of this…
According to a story from Oncology Nurse Advisor, a recent study found that patients with immune thrombocytopenia do not carry the same understanding as their doctors do when it comes…
According to a story from BioSpace, Incyte Corporation and Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has given the drug baricitinib Fast Track…
According to a story from news-medical.net, a team of researchers has found that patients who are more intellectually active can slow the neurodegeneration that appears in Huntington's disease. Such as…
According to a story from NTD, a former NFL cheerleader successfully diagnosed her own rare disease, pectus excavatum, after her symptoms were dismissed by her doctors as nothing more than…
According to a story from Cosmopolitan, a young woman who we will call Courtney (real name changed for privacy) was diagnosed with chronic fatigue syndrome. However, this diagnosis only occurred…
According to a story from philly.com, Markelle Fultz, who plays as a guard for the Philadelphia 76ers, was recently diagnosed with thoracic outlet syndrome. It has not been good news…
According to a story from Charcot-Marie-Tooth News, a group of scientists have developed a new scale that can be used to predict disease severity in infants that are diagnosed with…
According to a story from Debra of America, a new Phase 1 clinical trial being operated by Stanford University is currently recruiting participants. This trial will be a test of…
According to a story from Cornwall Live, parents Ben Keverne and Hannah Chapman were recently devastated by the loss of their daughter Maisie. The little girl was diagnosed with mitochondrial…
According to a story from Charcot-Marie-Tooth news, researchers have found that models of mutations affecting the mitofusin 2 protein (MFN2), which is the protein that is affected Charcot-Marie-Tooth disease type…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
