Exclusive iVeena speaker interview released for 3rd Ophthalmic Drugs Conference

18 - 20 November 2019, London, UK. Senior expert from iVeena is set to present at SMi's 3rd Annual Ophthalmic Drugs 3 Day Conference. Europe's leading Ophthalmic Drugs conference will explore new discoveries in the treatment of ocular rare disease, innovations in gene therapy, the challenges in drug delivery through a complex barrier, patient comfort and regulatory compliance which make up such core components within the Ophthalmic Drug sphere.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured Events Source Type: news

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Sarepta Therapeutics stock dropped as much as 13 percent Thursday after news emerged that a child participating in a trial of the company's experimental gene therapy for Duchenne muscular dystrophy experienced a serious health complication. Sarepta (Nasdaq: SRPT) has been testing a treatment for the rare disease that would insert a functioning gene into patients with the disease, who have a non-working copy of the gene. The Cambridge biotech's stock quickly dropped Thursday when it was reported…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
After years as a rare disease drugmaker, Sarepta Therapeutics Inc. is expanding into a neurological disease that affects millions of people worldwide and has been dominated for years by its Cambridge neighbor, Biogen Inc. Sarepta (Nasdaq: SRPT) said Wednesday that it's developing a new gene therapy treatment for multiple sclerosis, a disease in which the body's immune system attacks the coverings that protect nerves. That's the disease that fueled Biogen's rise to become the largest Massachusetts-based…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
CAMBRIDGE, Mass., June 25, 2019 -- (Healthcare Sales &Marketing Network) -- SmartPharm Therapeutics, a preclinical-stage pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of rare diseases, today anno... Biopharmaceuticals, Personnel SmartPharm Therapeutics, gene therapy
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
ConclusionDiamond –Blackfan anemia is a rare disease that carries significant morbidity and mortality if not diagnosed early and managed appropriately. Limited health resources, patient registries, and specialists as seen in developing countries result in a paucity of knowledge about Diamond–Blackfan anemia in Af rica. This case reminds clinicians about Diamond–Blackfan anemia as a cause for anemia in infants, the limitations in making the diagnosis in under-resourced health care systems, and the need for standardized treatment protocols applicable to resource-limited countries.
Source: Journal of Medical Case Reports - Category: General Medicine Source Type: research
A Chicago biotech claims in a new lawsuit that Cambridge venture capital firm Third Rock Ventures and bluebird bio CEO Nick Leschly worked to hinder its drug development efforts so it could more easily obtain a key part of its intellectual property. Errant Gene Therapeutics is suing Third Rock Ventures and Leschly three years after it sued New York-based Sloan Kettering Memorial Cancer Center over a stalled rare disease trial. In the lawsuit, filed on Friday in Suffolk County Superior Court, Errant…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
There ’s been an odd-sounding word circulating in the English headlines recently.The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation.   True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein[1]. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.Orkambi has been licensed by the EMAsin...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. ...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Conclusion This study described four Chinese LINCL siblings who were diagnosed by WES. The patients of these four families had similar disease courses started from motor regression or seizures to cognition regression and visual loss but carried mutations in different genes i.e. CLN2, CLN5, CLN6, and CLN7. The clinical features of LINCLs in these four Chinese siblings were not significantly different from those of Western patients. However, all Chinese LINCL patients in this study presented similar clinical course despite the affected genes. We assumed it as an ethnic specific clinical course according to our observation. ...
Source: Frontiers in Genetics - Category: Genetics & Stem Cells Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
AbstractRare diseases pose a global challenge, in that their collective impact on health systems is considerable, whereas their individually rare occurrence impedes research and development of efficient therapies. In consequence, patients and their families are often unable to find an expert for their affliction, let alone a cure. The tide is turning as pharmaceutical companies embrace gene therapy development and as serviceable tools for the repair of primary mutations separate the ability to create cures from underlying disease expertise. Whereas gene therapy by gene addition took decades to reach the clinic by increment...
Source: Molecular Diagnosis and Therapy - Category: Molecular Biology Source Type: research
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