Lennox-Gastaut syndrome (LGS) is a form of epilepsy. Most patients with this condition are resistant to current treatments and experience seizures throughout the duration of their life. They may face…
A recent study led by Morten Aagaard Nielsen and Tue Wenzel Kragstrup, who both work at Aarhus University in Denmark, hopes to help physicians determine the best personalized treatment for…
Roche has recently released the results of the latest phases of their study of gantenerumab, which is a treatment of a rare form of Alzheimer's disease. This form, which is…
Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease. This treatment has also received…
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on chromosome 10q26. Its discovery just…
According to a story from Targeted Oncology, findings from a recent phase 1 trial have discovered a new potential treatment for myelofibrosis. The study combined two different drugs: panobinostat (marketed…
Angiosarcoma Angiosarcoma is a rare condition caused by a tumor within the endothelial cells. These cells line the blood vessels in the body, and therefore the tumors can occur practically…
Neurogene, which works to provide treatments for rare neurological disorders, has recently joined with Behind the Seizure. Behind the Seizure was established by two other companies, BioMarin and Invitae, with…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
According to a story from gurufocus.com, the pharmaceutical company Zogenix, Inc., has recently announced top-line results from its phase 3 clinical trial. This trial is testing FINTEPLA® as a treatment…
An article in CURE® describes two extremely rare types of tumors: paraganglioma and pheochromocytoma. One in three thousand individuals may have either or both of these tumors. The symptoms accompanying…
As originally reported in Mayo Clinic, Dr. Veronique Belzil realized she wanted to leave her job as a psychologist to find a cure for amyotrophic lateral sclerosis when she saw…
According to a study published in the scientific journal Neurology, the survival rates of patients with Lambert-Eaton myasthenic syndrome (LEMS) were compared to that of the general population as well as…
According to a story from mmm-online.com, a recent infographic highlights the challenges that rare disease patients worldwide still face when it comes to getting the treatment that they so desperately…
NeuroSense Therapeutics, a biotechnology company, is in the process of developing PrimeC. PrimeC was created for the treatment of amyotrophic lateral sclerosis (ALS), and it is meant to slow or…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
Having a rare disease means you could have one of any of 5,000-7,000 distinct disorders, each with their own quirks. Just how many there are though remains unclear. As originally…
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Scientists in four continents worked for over a decade on a massive project that has resulted in the most detailed picture of cancer ever produced. Researchers now have a huge…
The National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) of the Food and Drug…
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients may be able to receive…
According to a story from curetoday.com, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal: finding better treatments for cholangiocarcinoma,…
NeuroQWERTY is a device created by nQ Medical that is meant to monitor brain health and the progression of the disease in those with Parkinson's. It has recently been granted…
Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…
As originally reported in StatNews, Mark Zuckerberg, founder of Facebook, along with his wife, Dr. Priscilla Chan, a former pediatrician and now head of the Chan Zuckerberg Initiative, made an…
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