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…
According to a story from scienmag.com, a pair of five year grants that were awarded to Dr. Mark Zylka will go towards projects focused on autism and the rare disease…
Are we ready for our close-up? The second annual Selections from DISORDER: The Rare Disease Film Festival at Biotech Week Boston will be held at the Takeda Auditorium in Cambridge,…
According to a story from The Boston Globe, the drug developer Ovid Therapeutics plans to move forward with a late stage clinical trial that will test the company's investigational drug…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
Angelman Syndrome (AS) is a rare genetic and neurological condition affecting roughly one person in every 12,000 to 20,000 individuals. People with AS often have learning difficulties, and struggle severely…
It feels good to rally together for a good cause, doesn't it? That must be the case for this Cedar Valley, Iowa community who answered the call when the mother…
Angelman Syndrome (AS) is a neurogenetic disorder. It's not fatal, but prevents patients' from living independently. It causes neurological impairment, seizures, and balance issues among other problems. It's caused by a…
Angelman syndrome impedes the brain's ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the…
According to a story from mirror.co.uk, Lee and Charmaine Taggart of Kent are the parents of Martha, a six year old girl who was born with Angelman syndrome. Martha's diagnosis…
The drug minocycline is a tetracycline antibiotic. Previous research using mouse models has indicated that it may have anti-inflammatory and antiapoptotic (prevents cell death) properties and may benefit some neurological…
The US FDA has awarded Orphan Drug status to the investigational drug NSI-189 for use by people with Angelman syndrome. You can read the source article here, at Nasdaq. About…
Angelman and Dup15q Syndromes: Shared Pathways to Discovery This event will consist of two days of scientific research presentations pertaining to Angelman syndrome and dup15q syndrome. The symposium will also…
According to a story from Angelman Syndrome news, Angelman syndrome was recently given its own disease unique disease code: Q93.51. The new code will help make researching and tracking the…
A new drug being developed to treat Angelman syndrome (AS) has just been approved for ‘orphan drug’ status in the U.S., reports GlobeNewswire. The drug, called GTX-101, works by activating the…
According to a story from pharmiweb.com, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation…
When Terry Jo Bichell learned her son was diagnosed with Angelman syndrome, but little knowledge existed on the disease, she decided to get her PhD and do research herself, reported…
Originally reported by Fox 17 Nashville, many Tennessee residents stress that a new tax reform bill will eliminate continued drug advancement and development for many rare diseases. The House and…
Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…
Rare disease began shaping acclaimed photographer, Karen Haberberg's family years before she was born. Her parents had given birth to her older brother, who was diagnosed with Tay-Sachs. Tay-Sachs is a…
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