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The global economic landscape has been fraught with uncertainties due to the ongoing pandemic, geopolitical tensions, and inflationary pressures. Amidst this backdrop, Chinese concept stocks have managed to stand out as beacons of resilience and growth. Companies listed on US exchanges such as Alibaba, Tencent, and JD.com have witnessed significant gains in their stock prices, outpacing the performance of popular US indices like the S&P 500 and the Nasdaq.Even teams like Chelsea and Manchester City, known for their big-money signings, have found themselves leaning on their veteran players to grind out results. Players like Cesar Azpilicueta and Fernandinho have provided stability and composure in defense and midfield, respectively, ensuring that their teams remain in contention for silverware.
Ariana Grande, Cynthia Erivo go viral in ‘Wicked’ press tour Ariana Grande and Cynthia Erivo send fans wild after trending moment Ariana Grande held on to Cynthia Erivo’s long fingernail in an interview this week and the viral clip has been making rounds on social media. During an interview on Tuesday, November 19th, the Wicked co-stars were told that several queer fans on social media were "taking the lyrics of Defying Gravity and really holding space with that and feeling power in that." "I didn't know that was happening," Erivo, 37, told Out Magazine . "That's really powerful. That's what I wanted," she added, looking over at Grande, 31. "I think that it's really important for [Elphaba] in that moment to not allow the things that have hurt her, that have stripped her of her humanity, to keep her down," she continued, referring to her character’s musical. While she was talking, the Disney alum gently took hold of her long, green fingernail, which was a nod to her character Elphaba and lightly tapped it, as a gesture of compassion and support. Fans had a lot to say about the gesture and it went viral over social media. One wrote, "I've officially been thinking about ariana holding cynthia’s nail for a full day, I see this image every time I close my eyes." Another chimed in, "I genuinely cant stop laughing at this. Every time I see it I burst into giggles." A third wrote, "So haunted by the image of Ariana Grande reaching out a trembling hand to grasp Cynthia Erivo’s single finger." Another found themselves “mesmerised” by the video, “I’m completely mesmerized by this clip ... it’s almost like they are speaking a language I don’t understand, I can’t catch the sense of it for a single moment. I’m not understanding the words or the feelings or the energy at all. It’s amazing." Wicked came out on Friday in theatres. Kanye West faces legal trouble for Diddy party accusations Adele speaks out on ‘Wicked’ plans ahead of concluding residency Liam Payne’s shocking footage before death raise concern Stephanie Beatriz reveals her father’s death moulded her new roleEditorial: Legislation to keep kids safe online cannot wait
BOSTON--(BUSINESS WIRE)--Dec 8, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVYTM (exagamglogene autotemcel) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first and only approved CRISPR/Cas9 gene-edited therapy. The results, presented at the American Society of Hematology (ASH) Annual Meeting and Exposition, continue to demonstrate the transformative, durable clinical benefits of CASGEVY. The longest follow up for both SCD and TDT patients now extends more than 5 years, with a median of 33.2 months and 38.1 months, respectively. “These comprehensive data provide additional evidence of the benefits of eradicating transfusion requirements for people with transfusion-dependent beta thalassemia and vaso-occlusive crises for those with sickle cell disease,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair of Vertex’s TDT Program Steering Committee, and Presenting Author of the CASGEVY clinical data at ASH. “With median follow-up around three years there is strong evidence for the durability of these beneficial effects following treatment with CASGEVY.” “CASGEVY is changing the outlook for people living with sickle cell disease and beta thalassemia, with these data reinforcing the immense clinical value a durable one-time therapy can provide to patients,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We have a strong commitment to build on our progress in bringing CASGEVY to patients around the world.” New long-term follow-up data presented from the CASGEVY trials Vertex had seven abstracts accepted at the ASH annual meeting as outlined below: Progress in bringing CASGEVY to patients around the world CASGEVY is approved for both SCD and TDT in the U.S., the European Union, Great Britain, Canada, Switzerland, Bahrain and the Kingdom of Saudi Arabia, and Vertex plans to make submissions in the United Arab Emirates and Kuwait. More than 45 authorized treatment centers have been activated globally to support the delivery of CASGEVY, and more than 40 patients have had a first cell collection. Vertex is continuing to work with reimbursement authorities to secure sustainable access for patients. Through this work, Vertex has agreements to provide CASGEVY in multiple countries, including the U.S., England (TDT), Austria, Bahrain and the Kingdom of Saudi Arabia, and continues to make strong progress in others, including positive Health Technology Assessments (HTAs) in Canada for both diseases and advancing access negotiations for SCD patients in England. In the U.S., Vertex recently secured an industry-first, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS) on a single outcomes-based arrangement available to all state Medicaid programs to ensure broad and equitable access to CASGEVY. To support this progress on patient access and growing patient demand, Vertex has received approval for a third manufacturing facility for CASGEVY with our partner Lonza. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive and life-shortening disease. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the U.S. of managing SCD for patients with recurrent VOCs is estimated between $4 and $6 million. SCD is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is VOCs, which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires a lifetime of treatment and results in a reduced life expectancy. In the U.S., the median age of death for patients living with SCD is approximately 45 years. A cure for SCD today is a stem cell transplant from a matched donor, but this option is only available to a small fraction of patients living with SCD because of the lack of available donors. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the U.S. of managing TDT are estimated between $5 and $5.7 million. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In the U.S., the median age of death for patients living with TDT is 37 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of people living with TDT because of the lack of available donors. About CASGEVYTM (exagamglogene autotemcel [exa-cel]) CASGEVYTM is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CLIMB Trials The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY (exagamglogene autotemcel) WHAT IS CASGEVY? CASGEVY is a one-time therapy used to treat people aged 12 years and older with: • sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs • beta thalassemia (β-thalassemia) who need regular blood transfusions CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia. IMPORTANT SAFETY INFORMATION What is the most important information I should know about CASGEVY? After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels. You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects. How will I receive CASGEVY? Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment. After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment. STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week. During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY. STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY. STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time. After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home. What should I avoid after receiving CASGEVY? What are the possible or reasonably likely side effects of CASGEVY? The most common side effects of CASGEVY include: Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms: These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of CASGEVY Talk to your healthcare provider about any health concerns. Please see full Prescribing Information including Patient Information for CASGEVY. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn , Facebook , Instagram , YouTube and X . (VRTX-GEN) Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Franco Locatelli, M.D., Ph.D. and Carmen Bozic, M.D., in this press release, and statements regarding expectations for the anticipated transformative, durable clinical benefits of CASGEVY, plans to continue working with reimbursement authorities to secure sustainable access for patients, including our expectations for progress in Canada and England, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that eligible patient access to CASGEVY may not be achieved on the anticipated timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com . You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. View source version on businesswire.com : https://www.businesswire.com/news/home/20241206536804/en/ CONTACT: Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.comMedia : mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 KEYWORD: MASSACHUSETTS EUROPE UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: BIOTECHNOLOGY PHARMACEUTICAL HEALTH CLINICAL TRIALS SOURCE: Vertex Pharmaceuticals Incorporated Copyright Business Wire 2024. PUB: 12/08/2024 12:52 PM/DISC: 12/08/2024 12:50 PM http://www.businesswire.com/news/home/20241206536804/en
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Additionally, the team's defensive frailties have been a cause for concern, with Manchester United conceding goals at an alarming rate. The lack of solidity at the back has resulted in the team losing valuable points and slipping down the table, further highlighting their struggles this season. Without a strong defensive foundation, it has been difficult for the team to compete at the highest level and challenge for silverware.As the closed beta testing for "From The Forgotten" is set to kick off on December 20th, anticipation is building among eager gamers who are eager to get their hands on this exciting new title. Players are encouraged to sign up for the beta test and experience firsthand the adrenaline-pumping gameplay, strategic depth, and competitive thrill that "From The Forgotten" has to offer.
Furthermore, Europe should strive to promote values such as sustainability, human rights, and environmental protection in its partnership with China. By advocating for responsible business practices and corporate social responsibility, Europe can help shape a more ethical and sustainable global economy.
Kansas once required voters to prove citizenship. That didn't work out so wellOne of the standout designs from the collection is a sleek black jersey with bold gold accents, reminiscent of the Los Angeles Lakers' colors that Kobe Bryant famously wore throughout his illustrious career. The jersey features intricate detailing inspired by Bryant's Mamba Mentality, with snakeskin patterns cleverly integrated into the fabric to symbolize his fierce competitiveness and unwavering determination. The iconic Ballhalo logo sits proudly on the chest, a symbol of the enduring legacy that Kobe left behind in the world of sports.Wisconsin high school football: Grantsburg bows in Division 6 state final