Tag Archives: 杭州楼凤

CHOP celebrates EC approval of first and only gene therapy for patients

first_imgReviewed by James Ives, M.Psych. (Editor)Dec 6 2018Children’s Hospital of Philadelphia (CHOP) celebrates a pivotal moment in medicine: approval by the European Commission (EC) of LUXTURNA® (voretigene neparvovec), the first and only gene therapy for patients with an inherited retinal disease, last month. This also makes LUXTURNA the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and European Union (EU).The EC approved LUXTURNA, a one-time gene therapy for the treatment of vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations, in pediatric and adult patients who have sufficient viable retinal cells. RPE65 -mediated inherited retinal disease is a progressive condition that leads to total blindness in most patients.The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway. In December 2017, the U.S. Food and Drug Administration (FDA) approved LUXTURNA for use in patients in the U.S.”The European Commission’s approval of LUXTURNA highlights the vital role of pediatric research in developing breakthrough cures,” said Bryan Wolf, MD, PhD, Chief Scientific Officer and Chair of the Department of Biomedical and Health Informatics at Children’s Hospital of Philadelphia. “The research conducted as a collaborative effort between CHOP’s Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) and investigators at the Perelman School of Medicine at the University of Pennsylvania laid the groundwork for this revolutionary gene therapy, which was developed and is now manufactured by Spark Therapeutics. Today, we are thrilled to see LUXTURNA approved as a therapy for children and adults outside the U.S.”Related StoriesGuidelines to help children develop healthy habits early in lifeResearchers identify gene mutations linked to leukemia in children with Down’s syndromeRevolutionary gene replacement surgery restores vision in patients with retinal degenerationCHOP founded Spark Therapeutics in 2013 in an effort to accelerate the timeline for bringing new gene therapies to market. Spark’s mission, to create a world where no life is limited by genetic disease, was to build on the foundational research conducted over a multi-year period by the CHOP and Penn Medicine teams.Beginning in 2000, the initial research for LUXTURNA was conducted by Jean Bennett, MD, PhD, F.M. Kirby professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania’s Scheie Eye Institute, and Albert M. Maguire, MD, a professor of Ophthalmology at Penn’s Perelman School of Medicine and an attending physician at CHOP. Bennett and Maguire joined forces with then-CHOP researcher Katherine A. High, MD, a gene therapy pioneer who directed the CCMT and who is now Spark’s President and head of research and development. Dr. Maguire served as a Principal Investigator of the therapy’s clinical trials.In the U.S., the gene therapy is currently administered at 10 treatment centers by leading retinal surgeons who receive training provided by Spark Therapeutics on the administration procedure.In January 2018, Spark Therapeutics entered into a licensing and supply agreement with Novartis covering development, registration and commercialization rights to LUXTURNA in markets outside the U.S. Upon the transfer of the marketing authorization from Spark Therapeutics to Novartis. Novartis can commercialize LUXTURNA in the EU/EEA. Novartis already has exclusive rights to pursue development, registration and commercialization in all other countries outside the U.S., and Spark Therapeutics will supply the gene therapy to Novartis.Source: https://www.chop.edu/news/children-s-hospital-celebrates-european-commission-approval-first-its-kind-gene-therapylast_img read more

10 million endowed gift supports psychosis research efforts

first_imgRelated StoriesSchwann cells capable of generating protective myelin over nerves finds researchTargeted treatment offers symptom relief for patients with psychosisStudy: Treatment of psychosis can be targeted to specific genetic mutationBecause the gift is endowed, the perpetual funding will secure the future of behavioral health research at Vanderbilt.”We are already providing clinical care to many patients during their first episode of psychosis. This transformational gift now allows us to map the trajectory of psychosis,” said Stephan Heckers, MD, MSc, chair of the Department of Psychiatry and Behavioral Sciences. “The outstanding translational research environment at Vanderbilt University Medical Center is the ideal place to realize the vision of our donor.”The gift will allow researchers to engage and track patients for longer periods of time, enabling longitudinal research studies that could otherwise not be accomplished. Through enhanced assessments, increased medication monitoring and earlier interventions, the team at Vanderbilt hopes to gain insight into what determines positive and negative treatment outcomes and to extend the lives of people with psychotic disorders, which are associated with a shortened life expectancy.”We are extremely grateful to the donor for making this translational research possible. The donor’s vision will impact many patients at Vanderbilt and will greatly advance our research agenda,” said Heckers. Reviewed by Kate Anderton, B.Sc. (Editor)Jun 13 2019Vanderbilt University Medical Center’s Department of Psychiatry and Behavioral Sciences has received a $10 million endowed gift to fund translational research, support clinical programs and create an endowed chair within the department.As a family member of a loved one with schizophrenia, the donor, who wishes to remain anonymous so the focus remains on the patients and new treatments, is energized by the potential of developing early treatments to slow or prevent psychosis from reaching more critical stages.”Seeing firsthand the devastation of the effects that these diseases have on not just the individual, but the whole family, I believe we need to try anything possible to help alleviate the pain of these experiences,” said the donor. “We keep hoping that something will come along that helps slow the progression of psychotic disorders. If we can work with patients through their first episodes so we can hopefully prevent future ones, that will be the key to improving lives.”The donor made the gift to help other families find answers as they struggle through the challenges of learning a loved one has a psychotic disorder and finding viable treatment options. The early days can be difficult. The burden of psychosis ripples through families as uncertainty and difficulty alter the fabric of life. We are grateful for the pivotal generosity that will ensure other people, and their families, are supported and better understood as they tackle treatment. This gift will reverberate for decades to come.”Jeff Balser, MD, PhD, President and Chief Executive Officer of Vanderbilt University Medical Center and Dean of Vanderbilt University School of Medicinecenter_img Source:Vanderbilt University Medical Centerlast_img read more

Videoondemand and the myth of endless choice

first_img Film industry eyes Internet future at Venice fest If you like independent, art-house films or other specialised movies, you may have heard of the Romanian comedy-drama Sieranevada, which was released in 2016. The film was formally premiered as part of the main competition programme of the prestigious Cannes Film Festival and was subsequently shown at other international film festivals, including Toronto, New York and London. Due to its success on the festival circuit, Sieranevada was reviewed by 48 international film critics, and received a positive rating from 92% of them. Among these were UK-based trade journals, such as Sight and Sound and Screen International as well as mainstream newspapers The Guardian and The Telegraph. But while this publicity generated audience interest in the film, it has yet to secure distribution that would allow UK audiences to actually watch the film – it’s not in cinemas, on DVD/Blu-ray, nor on online video-on-demand platforms (VOD).The development of VOD has provided new opportunities for films to reach audiences. In particular, specialised films with traditionally limited distribution opportunities have taken advantage of this development. But are online audiences presented with an endless choice? Not really. So why is this?The digital film revolutionIn the mid-2000s, digital utopians such as Chris Anderson were already arguing that an endless choice of specialised and niche content would become available to online audiences. And more than a decade later, it’s true that distribution opportunities have increased for such content in the online market. Film audiences are able to browse through catalogues on transactional VOD platforms such as Amazon Video, Microsoft and iTunes where they can find tens of thousands of films. Provided by The Conversation Why can’t we get everything?In the film business, sales companies have an important role to play in the process of enabling access for films because they negotiate distribution deals with a range of distributors in international markets. But if sales companies are unable to sell distribution rights, they retain control over the distribution and release of those films.The development of the online market, in this respect, has opened up opportunities for them to work directly with VOD platforms or with content aggregators, who work as intermediaries between rights holders and VOD platforms. Examples of such content aggregators include The Movie Partnership, Juice Worldwide and Gunpowder & Sky.For instance, the comedy-drama Dreamland (2016), directed by Robert Schwartzman, premiered in the US Narrative Competition of the Tribeca Film Festival. The US sales company FilmBuff (now named Gunpowder & Sky) acquired worldwide distribution rights. In the UK market, the film was not released in cinemas or on DVD or Blu-ray, but FilmBuff made it available in the online market through direct connections with Microsoft and iTunes rather than via a UK distributor.Despite such opportunities, sales companies do not always work with content aggregators or directly with VOD platforms to make specialised films available if they are not picked up by distributors. Making films available online requires organisational effort and a low-cost investment in digital formatting, but returns on investment can be very modest. That explains why some films remain inaccessible to audiences – as demonstrated for the UK market in the table below. This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only. A selection of films released by distributors in the UK market. BFI weekend box office figures, IMDb, Amazon, Microsoft, iTunes, Author provided Endless choiceThe politics behind the process of providing access to specialised films ultimately affects producers and audiences. In the new digital economy of attention, producers demand wider distribution for their films, while audiences demand endless choice.This issue needs to be resolved. First, it needs to be addressed in film industry discussions between film producers and sales companies. In particular, sales companies should make a stronger commitment to making films available on transactional VOD platforms.Second, policymakers can intervene in the process of making specialised films available online. Public funding agencies, such as the British Film Institute (BFI) in the UK, provide substantial financial support for the production of specialised films. They can provide more distribution incentives to support cultural diversity in the online market for films in the UK. This would help to support greater cultural diversity, democratisation of access to films and enhancement of consumer choice. While cinema goers have always had limited options when it comes to the number of screens they can see their favourite art house movie on, the internet era was supposed to bring with it an endless choice. But what is becoming clear is that this utopian dream is still far from being realised. But there is still a significant proportion of films that remain inaccessible for audiences, even if – like Sieranevada – they have been selected for prestigious international film festivals.center_img This article was originally published on The Conversation. Read the original article. Explore further Number of specialised films released in the UK market. BFI weekend box office figures, Amazon, Microsoft, iTunes, Author provided What is available?In an effort to identify the proportion of well-regarded specialised films that reach audiences in the UK market, I analysed a sample of 119 such films shown at prestigious European and US film festivals in 2016. My analysis in the graphic below confirms that the online market creates distribution opportunities for a greater number of films than the theatrical cinema and DVD/Blu-ray markets:88 specialised films (74%) were given an online release on Amazon, Microsoft or iTunes71 specialised films (60%) were given a DVD/Blu-ray release61 specialised films (51%) were given a theatrical cinema releaseBut while access to specialised films has increased, 26% of specialised films remain inaccessible for audiences on any format. That is a remarkably high percentage – given that it is relatively easy to secure online access for films. My analysis includes a sample of specialised films selected for some of the most prestigious festival programmes, but it is likely that online availability is more limited among specialised films selected for less prestigious competitions. So why can’t online audiences see any film they want? It’s to do with the way the industry works. Credit: Shutterstock Citation: Video-on-demand and the myth of ‘endless choice’ (2018, August 16) retrieved 18 July 2019 from https://phys.org/news/2018-08-video-on-demand-myth-endless-choice.html Example of Amazon’s transactional VOD platform as of July 6, 2018 (Amazon’s catalogue includes 50,000 films). Credit: Amazon.co.uk, Author providedlast_img read more

HALs exclusion from deal was UPAs failureHALs exclusion from deal was UPAs

first_imgDefence Minister Nirmala Sitharaman   –  THE HINDU COMMENTS Defence Minister Nirmala Sitharaman on Tuesday sought to turn the tables around, blaming the previous Congress-led UPA regime for keeping State-run Hindustan Aeronautics Ltd (HAL) out of the Rafale fighter jet deal.The Congress has been accusing the Narendra Modi government of ignoring the public sector unit in the manufacture of the combat aircraft, and favouring the Anil Ambani’s Reliance Defence.Sitharaman claimed the price negotiated for the Rafale by the Modi government was nine per cent cheaper than the UPA deal.“One of the things that did not happen during the UPA was that between Dassault Aviation and HAL they could not agree on production terms. That is why they could not go together. So that speaks for which government failed in terms of getting them together,” said Sitharaman, speaking at an interaction organised by the Indian Women’s Press Corps on Tuesday.Sitharaman was responding to questions based on Congress leader and former defence minister AK Antony’s allegations that if the UPA deal wasn’t cancelled by this government, HAL would’ve acquired the state-of-the-art technology through the transfer of technology and would’ve gained experience to manufacture fighter aircraft.“The previous government could have done anything to strengthen HAL’s offer to ensure that HAL’s terms were appealing enough for Dassault to conclude the deal with it. So the entire issue of HAL not being chosen happened during there time. “You (UPA) have not taken care of HAL. You have not made the terms appealing enough for HAL to bid successfully with Dassault. You have not finalised buying of Rafale aircraft for depleting squadron of IAF,” she said, stressing that questions should be posed to the Congress on HAL.The Congress has accused the Modi government of causing a loss of ₹41,000 crore to the public exchequer by purchasing Rafale aircraft from the French government with the same configuration at thrice the price.Responding to allegations on the pricing front, the Minister said, “We have responded saying your basic price and the basic price that I am getting at, when compared with all the escalation and other things, is 9 per cent cheaper.” Defence Minister claims Modi govt’s procuring the jets 9% cheaper Published on ministers (government) September 18, 2018 COMMENT SHARE SHARE EMAIL SHARE defencelast_img read more

37 trains cancelled 16 diverted day after Amritsar tragedy37 trains cancelled 16

first_imgrailway accident Published on The railways cancelled 37 trains and diverted 16 trains on Saturday, a day after Dussehra revellers were mowed down by a train while they were watching the burning of a Ravan effigy while standing on the tracks near Amritsar, officials said.Sixty-one people were killed and more than 70 injured in the tragedy. Giving details, the railways said 10 mail/express trains and 27 passenger trains were cancelled. While 16 trains were diverted and reached their destination through a different route, 18 trains were short terminated, Northern Railways spokesperson Deepak Kumar said.The route between Jalandhar and Amritsar was suspended, he said. The railways maintains it had no information about the Dussehra function held near the tracks. Chairman Railway Board Ashwani Lohani said in a statement that the accident occurred at a midsection between the Amritsar and Manawala stations and not at a level crossing. “There was no information and no permission sought from us. The event took place at a place adjoining the railway land in private property,” he said.“At midsections trains run at their assigned speeds and people are not expected to be on the tracks. At midsections there is no railway staff posted. We have staff at the level crossings whose job is to regulate traffic,” he added, explaining why the railways was not alerted about the congregation by its staff.According to Lohani, the gateman was 400 metres away at a level crossing. If the driver had applied emergency breaks, there could have been a bigger tragedy, he said.The train was running at assigned speed and initial reports suggest that the driver applied brakes and the train slowed down, the top official said.Refusing to assign any blame, Lohani, who visited the spot at midnight, said the the national transporter has been carrying out campaigns exhorting people not to trespass. “We will take that forward,” he said. COMMENT SHARE SHARE EMAILcenter_img October 20, 2018 SHARE COMMENTSlast_img read more

Air strikes on Syrian rebel enclave intensify monitor

first_img Related News BEIRUT (Reuters) – Air strikes targeted rebel-held cities in northwest Syria on Friday, a war monitor reported, widening bombardment of the last major insurgent enclave to areas that had mostly escaped it. The strikes killed three people in Idlib and three in Maarat al-Numan, two of the largest cities in the region, the Britain-based Syrian Observatory for Human Rights reported. Another nine people were killed elsewhere in the enclave, it said. President Bashar al-Assad’s government, backed by Russia, has waged a major assault against the northwestern enclave since the end of April in a battle the Observatory says has killed nearly 2,450 people. Related News World 10 Jul 2019 Assad hits a wall in Syrian war as front lines harden {{category}} {{time}} {{title}}center_img Syria’s eight-year war has killed half a million people, driven half the pre-war population of 22 million from their homes, inspired a wave of militant attacks around the globe, and dragged in regional and world powers. Aid agencies say the scale of bombardment in the north west risks further humanitarian disaster. Hundreds of thousands of people have already fled towards the Turkish border. While both Idlib city and Maarat al-Numan were heavily targeted by airstrikes in earlier phases of the war, they have not been a major focus of bombardment in recent months, with the military more focussed on hitting areas near the front lines. There was a surge of violence this week on the area of Tel Hamamiyat on the southwestern edge of the enclave, where pro-government al-Watan newspaper and rebel statements reported major fighting. A spokesman for the local government controlling most of the enclave, which is held predominantly by jihadist factions, said Friday prayers had been suspended in several cities due to the air strikes. More than two months of Assad’s Russian-backed assault in and around Idlib province have yielded little or no military gains, marking a rare case of a campaign that has not gone his way since Moscow joined his war effort in 2015.Turkey, which backs some rebel groups in northwest Syria and controls an adjacent zone along its own border, agreed a truce last year with Russia to reduce warfare around the enclave. Those areas, and the quarter of Syria east of the Euphrates river which is held by U.S.-backed Kurdish groups, are the only parts still out of Assad’s control. (Reporting by Angus McDowall in Beirut and Sarah Dadouch in Istanbul; Editing by Andrew Cawthorne) World 07 Jul 2019 Russian-led assault in Syria leaves over 500 civilians dead – rights groups, rescuers World 10 Jul 2019 Exclusive: New chemical weapons team to launch first Syria investigationslast_img read more