Interim Six-Month Data of RPGR Gene Therapy Shows Significant Vision Improvement in Patients Living with X-Linked Retinitis Pigmentosa

Click to Access Audio Press ReleaseRARITAN, NJ, July 17, 2020 – The Janssen Pharmaceutical Companies of Johnson &Johnson announced today six-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP). The interim data showed that low and intermediate doses of the investigational adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR) were generally well-tolerated and indicated significant improvement in vision. Initial data on the novel AAV-RPGR asset, jointly developed with MeiraGTx Holdings plc, will be available today as a late-breaker, pre-recorded oral presentation at the American Society of Retina Specialists (ASRS) 2020 Virtual Annual Meeting.In patients with XLRP, the photoreceptors in the eye that are responsible for converting light into signals that are sent to the brain function poorly, leading to degeneration of the retina and legal blindness in adulthood. The companies’ AAV-RPGR gene therapy is being investigated to treat the most common and severe forms of XLRP caused by mutations in the RPGR gene by preserving and improving vision and slowing retinal degeneration. Currently, there are no approved treatments for this condition. “There is an urgent need to deliver a transformational therapy for people living with XLRP who experience progressive visual loss from childhood with eventual blindness in early adul...
Source: Johnson and Johnson - Category: Pharmaceuticals Tags: Innovation Source Type: news

Related Links:

Authors: Bergland OU, Søraas CL, Larstorp ACK, Halvorsen LV, Hjørnholm U, Hoffman P, Høieggen A, Fadl Elmula FEM Abstract PURPOSE: The blood pressure (BP) lowering effect of renal sympathetic denervation (RDN) in treatment-resistant hypertension shows variation amongst the existing randomised studies. The long-term efficacy and safety of RDN require further investigation. For the first time, we report BP changes and safety up to 7 years after RDN, compared to drug adjustment in the randomised Oslo RDN study. MATERIALS AND METHODS: Patients with treatment-resistant hypertension, defined...
Source: Blood Pressure - Category: Hematology Tags: Blood Press Source Type: research
Authors: Zhang W, Xu JZ, Lu XH, Li H, Wang D, Wang JG Abstract PURPOSE: We hypothesise that dietary sodium intake interacts with serum uric acid to influence blood pressure (BP) in children and adolescents. In the present study, we investigated ambulatory BP in relation to hyperuricaemia, dietary sodium intake and their interaction in children and adolescents with hypertension. MATERIALS AND METHODS:  A total of 616 study participants were 10-24 years old and had primary hypertension diagnosed after admission in a specialised inpatient ward. Ambulatory BP monitoring was performed during hospitalisat...
Source: Blood Pressure - Category: Hematology Tags: Blood Press Source Type: research
[Unilateral Irregularities in the Macular Pigment Epithelium in a 38-Year-Old Patient - "Acute Retinal Pigment Epitheliitis"]. Klin Monbl Augenheilkd. 2020 Oct 09;: Authors: Wykrota AA, Löw U, Fries FN, Seitz B, Abdin AD PMID: 33036057 [PubMed - as supplied by publisher]
Source: Klinische Monatsblatter fur Augenheilkunde - Category: Opthalmology Tags: Klin Monbl Augenheilkd Source Type: research
CONCLUSION: The proposed PHARMAC criteria will give access to these important drugs to those people with T2DM who will likely benefit the most. PMID: 33032305 [PubMed - in process]
Source: New Zealand Medical Journal - Category: General Medicine Tags: N Z Med J Source Type: research
Authors: Siamashvili M, Davis S Abstract INTRODUCTION: Bromocriptine mesylate quick release (QR) is a dopamine D2 receptor agonist and is the only oral, primarily centrally acting drug that can be used for the treatment of adults with type 2 diabetes. AREAS COVERED: The authors describe current recommendations on the use of bromocriptine mesylate QR. Major efficacy and safety parameters of the late phase trials, including The Cycloset Safety Trial, have been identified and presented. EXPERT OPINION: Efficacy of bromocriptine mesylate QR monotherapy appears to be low but is compensated by favorable safety pr...
Source: Expert Opinion on Pharmacotherapy - Category: Drugs & Pharmacology Tags: Expert Opin Pharmacother Source Type: research
Abstract Trace metals concentrations of 25 elements were determined for 22 subcomponents of biodegradable and non-biodegradable waste samples representing the United States municipal solid waste (MSW) stream collected during three separate waste sorts. The subcomponent trace metal concentrations and estimated composition results were used to predict trace metal concentrations present in the overall MSW stream along with MSW compost and waste to energy (WTE) ash, which were compared to health-based standards (i.e., US EPA regional screening levels) and to values previously reported in the literature. These estimate...
Source: Chemosphere - Category: Chemistry Authors: Tags: Chemosphere Source Type: research
Publication date: December 2020Source: Journal of Functional Foods, Volume 75Author(s): Tingting Wang, Lin Zheng, Tiantian Zhao, Qi Zhang, Zhitong Liu, Xiaoling Liu, Mouming Zhao
Source: Journal of Functional Foods - Category: Nutrition Source Type: research
TYPE 2 diabetes may feel like a minefield - can you eat this or that without spiking blood sugar levels? If you're feeling peckish, what's a good, healthy option for lunch?
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
CONCLUSIONS: Neuro-ophthalmologic findings are mostly normal in patients with visual snow syndrome. Retinal or neurological diseases must be excluded as possible causes of visual snow. PMID: 33029971 [PubMed]
Source: Journal of Clinical Neurology - Category: Neurology Tags: J Clin Neurol Source Type: research
CONCLUSIONS: Young adult IS patients in Korea exhibit low awareness and poor management of their risk factors. Although the short-term outcome was relatively favorable in those patients, having SLE was associated with unfavorable outcomes. More attention needs to be paid for improving awareness and controlling risk factors in this population. PMID: 33029967 [PubMed]
Source: Journal of Clinical Neurology - Category: Neurology Tags: J Clin Neurol Source Type: research
More News: Achromatopsia (ACHM) | Adenoviruses | Age-Related Macular Degeneration (AMD) | Allergy & Immunology | Biology | Blindness | Brain | Cancer & Oncology | Cardiology | Cardiovascular | Child Development | Clinical Trials | Diabetes | Endocrinology | European Medicines Agency (EMA) | Eyes | Food and Drug Administration (FDA) | Gene Therapy | Genetics | Health Management | Heart | Hospitals | Hypertension | Infectious Diseases | Learning | Legislation | Neurology | Neuroscience | Opthalmology | Pharmaceuticals | Profits and Losses | Pulmonary Hypertension | Retinitis Pigmentosa | Science | Study | UK Health | Universities & Medical Training | USA Health | Vaccines | Vitamin A | Websites