The London Marathon took place on April 23, 2023—and one mom was running for an especially important cause: to raise Angelman syndrome awareness. According to an article from the BBC,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
When Harper Webb was just six weeks old, her mother Johnna grew concerned about the girl’s health—call it mother’s intuition. Harper struggled with sleeping and feeding. Johnna began taking Harper…
The Foundation for Angelman Syndrome Therapeutics (FAST) recently entered a partnership with Rush University in order to establish a clinical trial and translational research center for rare neurodevelopmental disorders.…
Stat News recently published comments by Bruce Bloom, collaboration officer at Healx, Cambridge, UK heralding the major advances in the treatment and the study of rare disease during 2022.…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Missense mutations cause Angelman syndrome in around 5% of diagnoses. However, researchers have often been unclear or unsure of the biochemical consequences of these missense mutations on the affected…
February 15, 2013 marked the first-ever International Angelman Day. The day, still celebrated nine years later, aims to raise global awareness for Angelman syndrome. Additionally, International Angelman Day serves to…
In a news release from May 19, 2021, startup biotechnology company GeneTx Biotherapeutics LLC ("GeneTx") and biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared that the pair received Health Canada clearance…
In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics ("Biom") shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients…
Biotech company Ovid has released disappointing results from a trial of OV101, a potential treatment for Angelman's syndrome. The Phase 3 trial demonstrated that there was no significant difference in…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Ovid Therapeutics has recently announced that it will host a webinar in an effort to educate people about Angelman syndrome and their possible treatment for it, OV101. Speakers will focus…
According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration…
By Danielle Bradshaw from In The Cloud Copy Angelman syndrome is a rare genetic disorder that is a combination of multiple physical and mental disabilities. 1 in every 15,000 people…
A recent news release on BioSpace shares how GTX-102, an investigational antisense oligonucleotide drug therapy for Angelman syndrome, was granted Fast Track designation from the FDA. The therapy, created by GeneTx…
The FDA has recently granted GTX-102, a treatment for Angelman syndrome, the Fast Track designation. This designation means that the review process is expedited. It is meant specifically for drugs…
According to Angelman Syndrome News, researchers used rat models of Angelman syndrome to test gene editing as a potential therapy. They found that CRISPR technology deleted the defective UBE3A gene. However, rats…
According to a story from BioSpace, GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical, Inc. have recently announced that the first patient has been dosed in the companies' phase 1/2 clinical trial.…
Seizures are a common symptom of Angelman syndrome (AS) and the cause of these seizures was unknown for a long time. This lack of knowledge made it impossible to…
As originally reported in Angelman Syndrome News, Terry Jo Bichell is a PhD researcher, former director and scientific officer of the Angelman Biomarkers and Outcomes Measures Alliance and Tennessee's ambassador…
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…
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