The first patient has just been dosed in a Phase 3 trial of a drug to treat spinal muscular atrophy, reports Benzinga. The drug, called AVXS-101, is a gene therapy…
The National Center for Advancing Translational Sciences (NCATS) supports rare disease patients and their communities. They provide translational research funding, tools and other resources that are helping to address unique…
Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
According to a story from pharmavoice.com, a new partnership between the biotechnology company Vitrisa Therapeutics and the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) is in the works in order…
A study has just been published in the New England Journal of Medicine (NEJM) that found that the drug Brineura slows down the deterioration of language and motor function in…
A large clinical study will be carried out to develop a protocol for identifying a rare primary immunodeficiency disease called WHIM syndrome in patients, reports BioPortfolio. The Jeffrey Modell Foundation,…
According to a story from Harvard Health Publishing, a common problem with the upper chambers of the heart which cause the heart to pump inefficiently may be made less severe…
A clinical trial that gave children with progeria an experimental drug that was initially intended for cancer patients has produced promising results, reports The Washington Times. The study found that children…
According to a story from PR Newswire, The Marfan Foundation and Backpack Health are partnering so that the foundation can begin the creation of a patient registry for people affected…
A new study reported on by Hematology Times has found that being taught art can significantly decrease pain and anxiety, and promote a positive mood in patients with hematologic malignancies.…
The US Food and Drug Administration (FDA) has just awarded Breakthrough Therapy Designation to a drug (OMS721) being developed to treat thrombotic microangiopathy that has developed as a result of haematopoietic stem…
A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…
What if paranoia could be genetically located? A study of Prader-Willi syndrome gave scientists a chance to find out. Keep reading to learn more about this development, or follow the…
In St. Louis, Missouri, a local start-up is using novel research to make a big difference. The start-up, known as Unleash seeks to develop a unique cure for cancer. Keep…
A new device to treat incontinence is being developed by Peter Sayet at Precision Medical Devices Inc., reports the Orlando Sentinel. Incontinence is the term used when people pass urine…
A Phase 1 clinical trial on patients with cancers that are the result of a change to the tyrosine kinase receptor, also called RET, has produced encouraging results. Cancers caused…
Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…
A group of researchers led by Professor Gholson Lyon has discovered a genetic mutation that is linked to certain traits, reports Medical Xpress. The method used to discover it was…
According to a story from Neuroscience News, researchers have begun to test the effectiveness of a certain molecule in fighting against childhood brain cancers, such as medulloblastoma. It has the…
The charity organisation Stand Up To Cancer (SU2C) has just announced that $10 million is going to be given to a team of researchers to fund their work to improve…
A press release from Aprea Theraputics has just announced the results of a Phase II clinical trial of the drug APR-246 in patients with myelodysplastic syndromes (a group of blood…
According to a story from Cosmos Magazine, Dr. Kathryn North is committed to researching the vast expanse of the human genome in order to pinpoint the ways in which genes…
According to a story from the National Center for Advancing Translational Sciences, a group of scientists from the National Institutes of Health (NIH) has unearthed a new mechanism that could…
Researchers from Newcastle University in the UK have just developed a potentially life-saving device for patients with Addison’s disease, reports Newcastle University. Patients will be given a Quick Response (QR)…
A drug called Anavex 2-73 being developed to treat Parkinson’s disease dementia is planned to undergo testing in a Phase 2 clinical trial, reports Globe Newswire. Parkinson’s disease is a…
© Copyright 2024 Patient Worthy
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.
