According to a story from the Sydney Morning Herald, two-year-old William McLennan, son of Naomi Taylor and Ben McLennan, was diagnosed with spinal muscular atrophy about a year ago. The…
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 UK government is being petitioned to hold a referendum on the decriminalisation of the drug cannabis. This has implications for people with diseases that may be helped by using…
According to a story from philly.com, a recently released set of guidelines is placing new emphasis in the need for patients not to delay treatment during the early stages of…
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 gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…
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…
The Australian government will subsidize a new treatment for Hodgkin lymphoma, bringing its price down from $200,000 per course to $39.50 for most people, and $6.40 for concession patients, reports…
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…
When people are faced with cancer, their main concerns are understandably treatment and survival. However, many are shocked to discover that when they survive cancer, another sinister blow on their…
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…
The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension…
Imagine not being able to eat because you cannot swallow your food. Add to that intolerable pain and the inability to sleep at night. These are some of the symptoms…
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…
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…
On April 16th, global healthcare company Grifols announced its donation of 25 million international units (IU) of blood clotting factor drugs. These donations occurred within the last year, and are…
The US Food and Drug Administration (FDA) has just approved a new drug called Tavalisse™ to treat adults who have thrombocytopenia as a result of chronic immune thrombocytopenia (ITP), following…
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…
According to a story from edinburghnews.com, mother Karen Gray is about to seek access to cannabidiol oil for her five-year-old son, Murray, who has a rare type of epilepsy, Myoclonic Astatic…
© 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.
