The Munson family is fundraising to make changes to their home, as these adaptations will make life easier for their two children Oscar and his older sister Mary. This is…
Many little girls dream of being princesses, and Michele Kingsland is no different. Well, she is a little different, because her wish was actually granted. The Make-A-Wish Foundation crowned Michele…
In a recent news release, gene therapy company Taysha Gene Therapies ("Taysha") shared the publication of preclinical data on the company's drug candidate, TSHA-102. Currently, Taysha is exploring TSHA-102 as…
According to a story from Benzinga, there are at least eight drug companies that are currently working on a treatment for Rett syndrome, a rare genetic disorder. With no cure…
During clinical trials, Independent Data Safety Monitoring Boards (DSMBs) sometimes assess trial safety, trial integrity, and how well the trials are upholding patient needs. According to Business Insider, the DSMB…
Rett syndrome is a rare neurological disorder which often presents within the first 6 months to 1.5 years of a child's life. As the condition progresses, children often lose…
As the medical field expands, so do the options for new and intriguing medical research. According to Drug Target Review, researchers from the University of California (UC) San Diego…
Did you know that zebrafish, a tropical freshwater fish, are being more commonly used to model and study human diseases? Now, says Phys.org, researchers from the Garvan Institute of Medical…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…
Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
Scientists have found a cluster of genes that plays a role in the development of an autism subtype. The genes are involved in controlling brain development, as well as the…
What causes developmental disorders? How can the human genome play a role in diagnosing and treating these conditions? Well, a team of scientists from the Salk Institute recently set…
According to a story from Star Tribune, Chelsea Coenraads was 14 months old when her mother Monica became convinced that something seriously wrong with her daughter. She had failed to…
Recently, a team of Yale researchers wanted to understand how genetic mutations in patients with Rett syndrome resulted in neurological issues. Using a unique type of genetic testing, researchers were…
.jpg)
Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such…
As originally reported in Discover Magazine, Huda Zoghbi took an unexpected route to winning the Breakthrough Prize in Life Sciences in 2017 for her discoveries of the genes responsible for…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
.jpg)
ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of…
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…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…
© Copyright 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.
