While many patients are grateful for their doctors and treatments, how many know why they are so passionate about what they do? In an interview with Dr. Patrick Flume,…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
As sourced from QZ, trials for new medications are notoriously tedious. Rare disease populations know more than most that the barriers to meeting the entry criteria of a trial can…
According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…
According to a study published in January in the scientific journal Acta Neurologica Scandinavica, the use of gamma knife surgery as a treatment for a rare tumor called a vestibular schwannoma can…
According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug…
According to a story from The Columbus Dispatch, a variety of disorders, such as autism, can cause issues with sensory processing. What this means is that people on the autism…
According to a story from Sleep Review, the drug company Jazz Pharmaceuticals plc has recently filed a New Drug Application (NDA) for its investigational treatment JZP-258, which is being developed…
According to a story from CureDuchenne, the biotechnology company Dyne Therapeutics recently announced that CureDuchenne Ventures has made an equity investment that will support the development of all new precision…
According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…
According to a story from Fierce Biotech, there have been some impressive advances in the treatment of cystic fibrosis recently. The latest milestone was in developing and approving the drug…
According to a press release on Global Newswire, Novartis has received a positive opinion on Cosentyx® from the Committee for Medicinal Products for Human Use (CHMP). This positive opinion…
Research findings can sometimes come from unexpected places. For example, according to Parkinson's News Today, the benefits and shortcomings of an investigative gene therapy were explored through the post-mortem analysis…
Cystic fibrosis is caused by a mutation in the gene CFTR. This gene involves a protein that affects the chloride on cells; without this chloride the surface of the…
According to a story from Pulmonary Hypertension News, the results of a recent phase 3 clinical trial have determined that patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) stand…
According to a story from People's Pharmacy, US Surgeon General Jerome Adams recently stated in a tweet that regular citizens should stop buying face masks because they are not useful…
Mother of two, Mina Manchester, has been navigating raising two children with an immunocompromising disorder for four years. It's this unique experience that makes her feel more prepared and…
According to a story from Financial Buzz, the biotechnology company Inflazome recently announced that it has completed a phase 1 clinical trial. This trial was testing the company's experimental treatment…
According to a story from Buzzfeed News, Sharon Greenstein, who lives with the rare autoimmune disease known as Sjögren's syndrome, can no longer find the drug that she uses for…
According to a story from stuff.co.nz, a two year old boy named Rhys from Auckland, NZ has spent much of his young life in the hospital. This is because he…
According to a story from BioPorfolio, a recent study first published in STEM CELLS Translational Medicine describes a unique stem cell procedure that has the potential to be a major step…
Current treatment for Huntington's disease only relieves symptoms, allowing for the progression of the disease to continue. UniQure, a company that works with gene therapies, aims to change this. They…
According to a story from Esmo, the results of a recent phase 2 clinical trial bode well for pediatric and adolescent patients with neurofibromatosis type 1 (NF-1), a rare genetic…
According to a story from mdmag.com, a phase 2 trial testing the drug avapritinib as a treatment for systemic mastocytosis is currently ongoing. The results so far suggest that the…
According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in medical theory, but only in…
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