Corderman family: 'There's a lot of hope' for rare disease treatment
David and Nancy Corderman ’s lives were quickly upended when two of their four children were diagnosed with a rare genetic condition called Usher Syndrome. Today, they help spread awareness of the disease. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 7, 2023 Category: Pharmaceuticals Authors: Grant Welker Source Type: news
Janssen Presents Study Results Showing Clinical Efficacy for TREMFYA ® (guselkumab) and Long-Term Safety Profile for STELARA® (ustekinumab) for Patients Living with Inflammatory Bowel Disease at Digestive Disease Week® 2022
SPRING HOUSE, PENNSYLVANIA, May 24, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced new data from the Phase 2 GALAXI 1 clinical trial of TREMFYA® (guselkumab) in adult patients with moderately to severely active Crohn’s disease (CD), and from three separate long-term pooled analyses of adult patients with ulcerative colitis (UC) and CD treated with STELARA® (ustekinumab).1,2,3,4 These data are being presented as oral and poster presentations and are among 29 Janssen abstracts presented during the Digestive Disease Week® (DDW) meeting taking place in person and virtually in San Di...
Source: Johnson and Johnson - May 24, 2022 Category: Pharmaceuticals Source Type: news
New research suggests in-womb gene correction
(Oregon Health& Science University) New research led by hearing scientists at Oregon Health& Science University suggests an avenue to treat and prevent intractable genetic disorders before birth. Researchers at the Oregon Hearing Research Center, working with mice, injected a specially designed synthetic molecule into the developing inner ear of fetal mice 12 days after fertilization. The study found that the technique corrected the expression of a mutated gene that causes Usher syndrome. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 5, 2020 Category: International Medicine & Public Health Source Type: news
New mutations causing inherited deaf-blindness have been discovered
(Sechenov University) A team of scientists from Sechenov First Moscow State Medical University (MSMU) together with their colleagues from leading scientific centers of Moscow and India described a number of genetic mutations causing Usher syndrome (inherited deaf-blindness). They found previously unstudied unique mutations in investigated DNA regions. The results of the study were published in the Ophthalmic Genetics journal. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 22, 2019 Category: International Medicine & Public Health Source Type: news
ViewRay picks up ex-Spectranetics CEO Drake, COO Matin | Personnel Moves – July 25, 2018
ViewRay (NSDQ:VRAY) said yesterday it tapped former Spectranetics head Scott Drake as its new chief executive officer and former Spectranetics COO Shar Matin as its new COO.
Before joining ViewRay, Drake served as CEO and prez of Spectranetics, which was acquired by Royal Philips (NYSE:PHG) in a deal that closed last August. Drake also serves as board chair for AtriCure and JustRight Surgical.
Newly appointed COO Matin also worked for Spectranetics, starting in 2007, and holding the COO position from 2014 until it was acquired by Philips.
“I am excited to join the ViewRay team as we drive growth and innovation to ...
Source: Mass Device - July 25, 2018 Category: Medical Devices Authors: Fink Densford Tags: Business/Financial News avitamedical Bonesupport Clinical Innovations google intentsolutions ohiomedical Olympus personnel-moves Procyrion royalphilips Spectranetics Corp. ViewRay Inc. Source Type: news
Case Western Reserve research advance may prevent a form of hereditary hearing loss
(Case Western Reserve University) A research advance co-led by Case Western Reserve University School of Medicine's Kumar Alagramam, PhD, may stop the progression of hearing loss and lead to significant preservation of hearing in people with Usher syndrome type III, a form of hereditary hearing loss linked to defects in the sensory " hair " cells in the inner ear. USH3 is caused by a mutation in the clarin-1 gene. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 26, 2017 Category: International Medicine & Public Health Source Type: news
What is Usher Syndrome?
Discussion
Over 7000 diseases are considered rare disorders according to the National Organization for Rare Disorders® (NORD, rarediseases.org). NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. They have information on about 1200 rare diseases on their website (https://rarediseases.org/for-patients-and-families/information-resources/rare-disease-information/).
Hearing loss (HL) is not rare and is a common problem across the ages. It affects 360 million people worldwide which is about 5% of the population. There are many causes of hearing loss an...
Source: PediatricEducation.org - October 23, 2017 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news
Usher syndrome: Gene therapy restores hearing and balance
(Institut Pasteur) Scientists from the Institut Pasteur, Inserm, the CNRS, Coll è ge de France, University Pierre et Marie Curie, and University Clermont Auvergne, have recently restored hearing and balance in a mouse model of Usher syndrome type 1G characterized by profound congenital deafness and vestibular disorders caused by severe dysmorphogenesis of the mechanoelectrical transduction apparatus of the inner ear's sensory cells. These findings open up new possibilities for the development of gene therapy treatments for hereditary forms of deafness. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 22, 2017 Category: International Medicine & Public Health Source Type: news
Young disabled people 'suffering from loneliness'
Newsbeat hears from two people who suffer from Usher syndrome, which affects hearing and sight. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 11, 2017 Category: Consumer Health News Source Type: news
A quarter of us 'avoid conversations with disabled people'
Newsbeat hears from two people who suffer from Usher syndrome, which affects hearing and sight. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 11, 2017 Category: Consumer Health News Source Type: news
Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder
(Johns Hopkins Medicine) In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher Syndrome, a genetic condition in humans characterized by partial or total hearing loss, dizziness, and vision loss that worsens over time. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 23, 2017 Category: Global & Universal Source Type: news
Gene Therapy Restores Hearing in Mouse Model Gene Therapy Restores Hearing in Mouse Model
Researchers have used a newly developed gene transfer vector to restore a high level of hearing in a mouse model of Usher syndrome, the most common genetic form of deaf-blindness. They describe their work in two recently published papers.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - February 14, 2017 Category: Consumer Health News Tags: Pathology & Lab Medicine News Source Type: news
First small molecule targeted therapy to mitigate hearing loss in usher syndrome type 3
The first small molecule targeted therapy for progressive hearing loss has been developed in a mouse model of USH3, an USH classified by progressive loss of hearing and vision starting in the first few decades of life along with variable balance disorder. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - April 26, 2016 Category: Science Source Type: news
Mother suffering from toxic shock syndrome caused by a TAMPON wakes from coma
Deborah Usher, 27, from Porthmadog, Wales, suffered from toxic shock syndrome - a rare bacterial infection caused by a tampon. When she awoke from a coma, she had severe memory loss. (Source: the Mail online | Health)
Source: the Mail online | Health - December 18, 2015 Category: Consumer Health News Source Type: news
Sisters born profoundly deaf with gradually deteriorating vision have Usher syndrome
India and Samira Cox have Usher Syndrome, a genetic disorder that saw them born profoundly deaf with deteriorating vision. But despite the diagnosis, the Sydney sisters remain positive for the future (Source: the Mail online | Health)
Source: the Mail online | Health - November 29, 2015 Category: Consumer Health News Source Type: news
