First patient treated in clinical study for gene therapy for Usher syndrome type 1B
On September 16, 2024, AAVantgarde Bio, a biotechnology company from Italy, announced that the first patient has been treated in a clinical study investigating the safety and efficacy of a […]
Long-term outcomes of cochlear implantation in Usher syndrome
New insights from research Recent research by Mirthe Fehrmann and colleagues at Radboudumc has provided significant new insights into the long-term outcomes of cochlear implantation (CI) in people with Usher […]
An important next step in USH2c research
Erwin van Wijk (Radboudumc) has recently received a substantial grant from the Foundation Fighting Blindness USA to create a large animal model in collaboration with the research groups of […]
What are the best biomarkers and endpoints for future trials?
NEW RESEARCH THANKS TO THE ‘USHER ACTIE RIJNSBURG’ FRIENDS GROUP Usher syndrome, characterized by sensorineural hearing loss and progressive Retinitis Pigmentosa (RP), presents a significant challenge for the medical community. […]
Patient and researcher: a dynamic duo once again cycling together
BUT NOW ON A RACING TANDEM WITH GEARS In a remarkable collaboration between patients with Usher syndrome and researchers from Radboudumc, a crucial next step is made possible in the […]
HOPE FOR USH1B PATIENTS
CLINICAL TRIAL BY AAVANTGARDE TO COMMENCE AAVantgarde, an international biotechnology company based in Italy and co-founded by Professor Alberto Auricchio, is dedicated to overcoming the limitations of adeno-associated virus (AAV) […]
jCyte Initiates Phase 3 Clinical Trial for RP Cell Therapy
Biotechnology company jCyte is gearing up to launch a phase 3 clinical trial in the United States for its jCell therapy, following a successful phase 2B trial and with approval […]
Foundation supports innovative research into retinal diseases
Development of a ‘retina-on-a-chip’ platform An important new study has been launched to offer hope to people suffering from hereditary eye diseases such as retinitis pigmentosa, Usher syndrome, macular degeneration, […]
Resumption of the clinical trial for RNA therapy for USH2a has been terminated
UPDATE September 27, 2023 In mid-August, we reported that ProQR Therapeutics, which had previously been forced to halt its two promising clinical trials, had found a new partner in Laboratoires […]
Who cleans up ‘the mess’ from the cones?
New research on the cause of blindness due to Usher syndrome A research team led by Dr. Ronald Roepman (www.roepmanlab.com) is conducting research at Radboudumc, Netherlands, to investigate the underlying […]
The very first adgrv1-zebrafish model has been presented!
Recently, an article was published in the journal Cells about the scientific research on USH2C. Merel Stemerdink and her colleagues at the Radboudumc have developed a zebrafish model with a […]
Sleep problems Usher patient appears to be a hallmark feature of the disease
Vision impairment is not the driver of fatigue and sleep problems It was always thought that fatigue and sleep problems in patients with Usher syndrome are the result of increased […]
Does this mutation cause blindness? It does, doesn’t, does!
Janine Reurink ends long lasted controversy with major implications for healthcare Initially there was no doubt a specific mutation in the USH2A gene caused the eye disease retinitis pigmentosa. Spanish […]
Two groundbreaking projects awarded
‘Moon Rocket Grant’ awarded to two groundbreaking projects At this moment, there is no treatment yet for Usher Syndrome, a disorder that globally 400.000 people suffer from. In order to […]
Study into the best approach of USH1B
Stichting Ushersyndroom (Dutch Usher Syndrome Foundation) announces with pride its financing of a study that will test the best approach for USH1B (gene) therapy by making use of, among others, […]
Tears as a source of information?
Stichting Ushersyndroom and Usher Syndrome Ireland finance research into USH1b Can tears provide information that may be important for improving diagnostics and monitoring in future treatments of USH1b patients? In […]
Research “Development of exon excision therapy”
This study aims at permanently removing specific exons, which include hereditary mutations when patients are concerned, from the DNA of the photoreceptors in the retina and/or the hair cells of […]
New research: testing gene therapy
With extra large vectors on mini retinas Stichting Ushersyndroom [ Dutch Usher Syndrome Foundation] is funding the majority of the new research “Genetic drugs preventing blindness due to loss of […]
Minigenes USH2A: General status
By Erwin van Wijk, lead researcher Radboudumc Errors in the code of the USH2A gene explain the development of Usher syndrome in about 50% of all patients. In addition to […]
Swim at night and take a nap during the day
Zebrafishes suffering from USH2A have a disturbed sleep rhythm Are patients suffering from Usher Syndrome so tired because of the huge efforts made in connection with their poor hearing and […]
Stichting Ushersyndroom [Dutch Usher Syndrome Foundation] Awards Grant to Usher III Initiative to Support Patient Database
A global Usher III patient (USH3) database for future clinical trials This year, the North-American foundation Usher III Initiative has taken preliminary steps towards collecting the information necessary to establish […]
A view on the RUSH2a study
In the international RUSH2a study of Jacque Duncan MD, University of California, San Francisco, 120 patients spread over nine different clinics are monitored for four years. This study includes only […]
Sepul Bio (Théa) Launches Phase 2b Clinical Trial of Ultevursen
FIRST PATIENT WITH USH2A-ASSOCIATED RETINITIS PIGMENTOSA TREATED Sepul Bio has announced that the first participant has been dosed in the LUNA study, a Phase 2b clinical trial investigating ultevursen […]