magical sea castle aegaion
None
SAN DIEGO , Dec. 13, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the U.S. Food and Drug Administration has approved CRENESSITYTM (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH), a rare, serious and lifelong genetic condition involving the adrenal glands. CRENESSITY, a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF 1 ) antagonist, is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production, allowing for glucocorticoid dose reduction. It is a breakthrough in the treatment landscape for classic CAH. "For the last three decades, Neurocrine Biosciences, together with our late founder, Wylie W. Vale , has conducted groundbreaking research uncovering the critical role of corticotropin-releasing factor and its receptor, CRF 1 , in the pathophysiology of congenital adrenal hyperplasia," said Kyle W. Gano , Ph.D., Chief Executive Officer, Neurocrine Biosciences. "The approval of CRENESSITY is a significant milestone for the CAH community, and we are grateful to the individuals who participated in our clinical trials, including their families and caregivers, and to the clinical investigators who helped advance a new therapy and class of medicines." "Patients and families struggle to achieve balance between managing the symptoms of CAH and the side effects or complications of treatment with high-dose steroids, which may impact quality of life," said Dina Matos , Executive Director, CARES Foundation. "We are grateful to Neurocrine Biosciences for engaging with our community throughout the drug development process to understand our needs and ultimately providing this new medication that can help reduce excess adrenal androgens and the need for high-dose steroid treatment for individuals living with CAH." CRENESSITY is expected to be commercially available in approximately one week. The medication will be provided through PANTHERx Rare, a specialty pharmacy, to centralize and simplify CRENESSITY prescription fulfillment. Neurocrine Biosciences is committed to supporting patients in obtaining treatment with CRENESSITY by offering Neurocrine Access Support, a free, comprehensive assistance program created for patients, caregivers and healthcare providers. It offers a range of options to make sure patients have everything they need to begin and continue taking CRENESSITY. A dedicated Care Coordinator, backed by a team, is available to help patients and caregivers navigate the insurance process and identify appropriate financial assistance options. Most patients will pay $10 or less per month for CRENESSITY*. For more information, visit www.NBIaccess.com/crenessity or call 1-855-CRNSITY (276-7489) Monday-Friday 8 am-8 pm ET . *Additional terms and conditions apply. CAHtalyst TM Clinical Program Overview: The FDA approval is supported by the largest-ever clinical trial program of classic CAH, the CAHtalyst Pediatric and Adult Phase 3 global registrational studies. CAHtalyst Phase 3 data results in pediatric and adult patients with classic CAH were published in The New England Journal of Medicine . "The clinical results across both CAHtalyst studies support the efficacy and safety profile of CRENESSITY and its ability to reduce the overproduction of adrenal androgens, allowing for a meaningful reduction in glucocorticoid dosage, while maintaining or enhancing control of these androgens," said Richard Auchus , M.D., Ph.D., Professor, University of Michigan Health, Principal Investigator. "Chronic treatment with supraphysiologic glucocorticoids can cause a number of short- and long-term health consequences, such as obesity, hypertension and osteoporosis, so the ability for patients with CAH to lower their glucocorticoid dose to a more physiologic level can have profound benefits." In both CAHtalyst studies, CRENESSITY enabled lower steroid doses and decreased androgen levels. Phase 3 CAHtalyst Pediatric Study: Phase 3 CAHtalyst Adult Study: CRENESSITY was well tolerated with few treatment-related adverse events in both CAHtalyst studies. Pediatric and adult patients taking CRENESSITY had no treatment-related serious adverse events. Adrenal insufficiency and crisis are risks of living with CAH that CRENESSITY does not address and can occur when a patient's GC dose is too low. In the CAHtalyst Pediatric study, there were no cases of adrenal crisis among patients taking CRENESSITY or placebo. In the CAHtalyst Adult study, two patients (1.6%) taking CRENESSITY experienced adrenal crisis. No patients on placebo experienced adrenal crisis. However, one patient (1.7%) on placebo experienced adrenal insufficiency. Patients should work with their healthcare provider to manage GC dosing while taking CRENESSITY. For more information about CRENESSITY, visit Crenessity.com . About Congenital Adrenal Hyperplasia Congenital adrenal hyperplasia (CAH) is a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal steroid hormones, such as cortisol, aldosterone and adrenal androgens, which are essential for life. Approximately 95% of CAH cases are caused by variants of the CYP21A2 gene that leads to deficiency of the enzyme 21-hydroxylase (21-OH). Severe deficiency of this enzyme leads to an inability of the adrenal glands to produce enough cortisol and, in approximately 75% of cases, aldosterone. Because individuals with CAH are still able to produce androgens, the unused precursors that would normally be used to make cortisol instead result in the production of excess amounts of androgens. If left untreated, CAH can result in salt wasting, dehydration and even death. Historically, exogenous glucocorticoids (GCs) have been used not only to correct the endogenous cortisol deficiency, but doses used are higher than cortisol replacement needed (supraphysiologic) to lower the levels of adrenocorticotropic hormone (ACTH) and adrenal androgens. However, GC treatment at high doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease and osteoporosis. Additionally, long-term treatment with high-dose GCs may have psychological and cognitive impact, such as changes in mood and memory. Adrenal androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as excess facial hair growth and menstrual irregularities, testicular rest tumors in males and fertility issues in both sexes. About The CAHtalystTM Studies The Phase 3 CAHtalystTM global registrational studies were designed to evaluate the safety, efficacy and tolerability of CRENESSITY in children and adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The CAHtalyst studies were the largest-ever clinical trial program in classic CAH, including 285 pediatric and adult patients. The CAHtalyst Pediatric study included 103 pediatric patients aged four to 17 years. The study tested two questions. The first question evaluated whether four weeks of CRENESSITY treatment could improve androgen control. The second question evaluated whether an additional 24 weeks of CRENESSITY treatment enabled customized glucocorticoid (GC) down-titration while androstenedione levels were maintained or improved. The CAHtalyst Adult study included 182 adult patients aged 18 to 58 years. Similarly, the first question of the study evaluated whether four weeks of CRENESSITY treatment could improve androgen control, and the second question evaluated whether an additional 20 weeks of CRENESSITY treatment enabled GC reduction to physiologic range while androstenedione levels were maintained or improved. Data from the CAHtalyst Phase 3 studies supported approval of CRENESSITY by the U.S. Food and Drug Administration in December 2024. The open-label extension treatment portions of both studies are ongoing. About CRENESSITYTM (crinecerfont) CRENESSITYTM is a potent and selective, oral corticotropin-releasing factor type 1 receptor (CRF 1 ) antagonist developed to reduce and control excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a non-glucocorticoid (GC) mechanism for the treatment of classic congenital adrenal hyperplasia (CAH). Antagonism of CRF 1 receptors in the pituitary has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH. The robust clinical study data demonstrate that lowering adrenal androgen levels with CRENESSITY enables lower, more physiologic dosing of GCs to replace missing cortisol. CRENESSITY comes in capsules and an oral solution. The capsule formulation is available in 50 mg and 100 mg doses. The oral solution is available as a 50 mg/mL strength formulation. For adults 18 years and older, the recommended dosage is 100 mg twice daily taken orally with a meal. For pediatric patients four to 17 years of age weighing less than 55 kg (121 lbs), the recommended dosage is based on body weight and is administered twice daily, taken orally with a meal. For pediatric patients weighing more than 55 kg (121 lbs), the recommended dosage is 100 mg twice daily taken orally with a meal. Healthcare providers can work with patients to determine the appropriate formulation for use depending on patient needs. Patients receiving CRENESSITY should continue GC therapy for cortisol replacement. Important Information Approved Uses CRENESSITY (crinecerfont) is a prescription medicine used together with glucocorticoids (steroids) to control androgen (testosterone-like hormone) levels in adults and children 4 years of age and older with classic congenital adrenal hyperplasia (CAH). IMPORTANT SAFETY INFORMATION Do not take CRENESSITY if you: Are allergic to crinecerfont, or any of the ingredients in CRENESSITY. CRENESSITY may cause serious side effects, including : Allergic Reactions. Symptoms of an allergic reaction include tightness of the throat, trouble breathing or swallowing, swelling of the lips, tongue, or face, and rash. If you have an allergic reaction to CRENESSITY, get emergency medical help right away and stop taking CRENESSITY. Risk of Sudden Adrenal Insufficiency or Adrenal Crisis With Too Little Glucocorticoid (Steroid) Medicine. Sudden adrenal insufficiency or adrenal crisis can happen in people with congenital adrenal hyperplasia who are not taking enough glucocorticoid (steroid) medicine. You should continue taking your glucocorticoid (steroid) medicine during treatment with CRENESSITY. Certain conditions such as infection, severe injury, or shock may increase your risk for sudden adrenal insufficiency or adrenal crisis. Tell your healthcare provider if you get a severe injury, infection, illness, or have planned surgery during treatment. Your healthcare provider may need to change your dose of glucocorticoid (steroid) medicine. Before taking CRENESSITY, tell your healthcare provider about all of your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Tell your healthcare provider about all the medicines you take , including prescription and over-the counter medicines, vitamins, and herbal supplements. The most common side effects of CRENESSITY in adults include tiredness, headache, dizziness, joint pain, back pain, decreased appetite, and muscle pain. The most common side effects of CRENESSITY in children include headache, stomach pain, tiredness, nasal congestion, and nose bleeds. These are not all the possible side effects of CRENESSITY. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088. Dosage Forms and Strengths: CRENESSITY is available in 50 mg and 100 mg capsules and as an oral solution of 50 mg/mL. Please see full Prescribing Information About Neurocrine Biosciences, Inc. Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids,* as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com , and follow the company on LinkedIn , X (formerly Twitter) and Facebook . (*in collaboration with AbbVie) The NEUROCRINE BIOSCIENCES Logo Lockup and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. CRENESSITY and CAHtalyst are trademarks of Neurocrine Biosciences, Inc. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from CRENESSITY for the treatment of classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency; the value and benefits CRENESSITY brings to patients with CAH; the ability of Neurocrine Biosciences to ensure patients have access to CRENESSITY; and whether the results from our clinical trials of CRENESSITY are indicative of real-world results. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of CRENESSITY; whether CRENESSITY receives adequate reimbursement from third-party payors; the degree and pace of market uptake of CRENESSITY; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; risks associated with the Company's dependence on third parties for development and manufacturing activities related to CRENESSITY, and the ability of the Company to manage these third parties; risks that additional regulatory submissions for CRENESSITY or other product candidates may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding CRENESSITY; risks that post-approval CRENESSITY commitments or requirements may be delayed; risks that CRENESSITY may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended September 30, 2024 . Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law. © 2024 Neurocrine Biosciences, Inc. All Rights Reserved. CP-CFT-US-0070 12/2024 View original content to download multimedia: https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-fda-approval-of-crenessity-crinecerfont-a-first-in-class-treatment-for-children-and-adults-with-classic-congenital-adrenal-hyperplasia-302331772.html SOURCE Neurocrine Biosciences, Inc.Putin says will carry out more Oreshnik missile tests in combat MOSCOW: Russian President Vladimir Putin said on Friday that Moscow would carry out more tests of the hypersonic Oreshnik ballistic missile in “combat conditions,” a day after firing one on Ukraine. “We will continue these tests, including in combat conditions, depending on the situation and character of the security threats posted to Russia,” Putin said in a televised meeting with military chiefs. The Kremlin boss also ordered the missile, which flies at speeds of Mach 10 -- 10 times the speed of sound -- to be put into serial production. Russia is developing similar advanced systems, he added. “We need to begin serial production. The decision... has in effect been taken,” Putin said, praising the “particular strength of this weapon and its power. “The weapon system that was tested yesterday is another faithful guarantee of Russia ́s territorial integrity and sovereignty,” he added. Putin claimed no other countries in the world had such missile technology. While he conceded other states would soon develop them, he said: “That will be tomorrow, after a year or two. But we have this system now. That ́s important.” His choreographed meeting with the defence minister and those in charge of developing the missile came at the end of a week that has seen the Ukraine conflict escalate rapidly. Putin said the firing of the Oreshnik missile was a direct response to Kyiv ́s forces using US- and UK-supplied missiles on Russian territory for the first time. In an address to the nation on Thursday, Putin said Russia reserved the right to fire missiles at military facilities in countries whose weapons are being used by Ukraine -- specifically the United States and Britain. Meanwhile, Ukraine ́s parliament has cancelled Friday ́s session, lawmakers said, citing the risk of a Russian missile attack on the district of Kyiv where government buildings are located.The move comes after several foreign embassies, including the US representative office, shuttered temporarily over the threat of a strike on Kyiv. “The hour of questions to the government has been cancelled,” Yevgenia Kravchuk, an MP from the ruling party told AFP. “There are signals of an increased risk of attacks on the government district in the coming days.” The cancellation also comes a day after Russian President Vladimir Putin issued a warning to the West by test-firing a new intermediate-range missile at Ukraine.
Article content PORT-AU-PRINCE, Haiti — Haiti’s health minister has been removed from his post, government officials told The Associated Press on Thursday, following a deadly gang attack on the largest public hospital in the capital, Port-au-Prince. The two government officials, who spoke on condition of anonymity due to the sensitive nature of the issue, said Health Minister Duckenson Lorthe will be replaced by Justice Minister Patrick Pelissier until a new health minister is found. Two journalists and a police officer were killed Tuesday as gang members burst into the General Hospital and fired indiscriminately at reporters who were there to cover the facility’s reopening. It was one of the worst attacks on Haitian media in recent memory. Seven other journalists were injured. Jean Frans Regala, a photographer who survived, said journalists had been invited to the hospital by the health ministry but there was little security at the site. “The fact that the minister of health invited us, you feel that preparations have been made already,” Regala told the AP. “When we made contact with a police unit, the police told us they were not aware of the event.” The health minister did not show up at the event, for reasons that have not been explained. RECOMMENDED VIDEO On Tuesday, Johnson “Izo” Andre, considered Haiti’s most powerful gang leader and part of the Viv Ansanm group of gangs that has taken control of much of Port-au-Prince, posted a video on social media claiming responsibility for the attack. The video said the gang coalition had not authorized the hospital’s reopening. Gang violence has worsened in Haiti since the nation’s president was killed in a 2021 coup attempt. Gangs are believed to control 85% of the capital and earlier this year staged attacks on the main airport and the country’s two largest prisons. The Caribbean country has struggled to organize an election that will restore democratic rule, and is currently governed by a transitional council made up of representatives of political parties, business groups and civil society organizations.
U.K. stocks higher at close of trade; Investing.com United Kingdom 100 up 1.38%
AP News Summary at 6:44 p.m. EST
Netflix getting set to air NFL on Christmas Day
NYSE American Suspends Trading of Golden Minerals' Shares
Ask Madison: Who was Victoria's 'Candy Man'?
Since the launch of Valve's , arguably the first major dedicated PC gaming handheld, other manufacturers have released their own takes on the PC gaming handheld like the , and . The Asus ROG Ally was the second major PC gaming handheld release, and was generally well-received, but how does it compare to the Deck on price, specs, and performance? Let's find out. Valve’s Steam Deck costs $399 / £349 / AU$649 for its base 256GB LCD model, and there are two additional models, which are $549 / £479 / AU899 for the 512GB version and $649 / £569 / AU$1,049 for the 1TB model. There are two models of the Asus ROG Ally with the entry-level version equipped with the Ryzen Z1 SoC, which currently retails for $399.99 (around £640 / AU$1,280), and the version with an AMD Ryzen Z1 Extreme selling for for $549.99 / £449 / AU$999. In terms of pricing, Steam Deck was the original the winner, though since the ROG Ally's launch in 2023 it's dropped in price considerably with plenty of deals bringing it down even lower. Right now, both systems are pretty even in price, so they tie in this regard. The original Asus ROG Ally is much smaller and lighter than the Steam Deck and other PC gaming handhelds., and its unique honeycomb white chassis made it stand out. Many gamers will be rightfully tempted to invest in it because of its far more lightweight and distinctive aesthetically. They both feature dual analog sticks, a 7-inch screen, and buttons in the front and back of the system. Both also have ventilation exhausts on the top of the system to compensate for the massive amounts of heat generated by their components. Their button layout and grips are also quite similar in size, feel, and quality. The Steam Deck is now slightly lighter and smaller, with an OLED model that upgrades the display even more. In terms of design, I would nominate the ROG Ally as the tentative winner as it's simply lighter and better looking. But if you're craving a simple and practical design, then the Steam Deck may be more your cup of tea. The obvious difference between the Steam Deck and Asus ROG Ally are its specs, as the latter is a far more powerful machine due to its superior CPU. This means that games look much cleaner and run better on the ROG Ally. While the ROG Ally isn't capable of the power gaming of its updated version, the ROG Ally X, it's still quite impressive in its own right. It's capable of smoothly running DirectX 11 and even DirectX 12 at times (though the latter can crash after reboot. I tested this out with the game and it ran almost completely stutter-free Shockingly, even the older Steam Deck model can run demanding games like , though you'll have to keep the settings down. And the OLED version can handle the game even better, with Medium and Low averaging around 26fps. The Steam Deck has a better OS, however, as the Armory Crate UI on the Ally is, quite frankly, just plain bad (and that's not even factoring in how poorly runs on a gaming handheld). Meanwhile, Steam's OS is built specifically for the system, so it's faster to start up and more intuitive to use. Performance-wise, the Steam Deck gets the win here. While gaming performance is better overall on the ROG Ally, the Steam Deck is more consistent and its OS is vastly superior. So which is the better PC gaming handheld? Surprisingly, both systems are pretty much tied at this point. The Steam Deck is still considered by many to be the best PC gaming handheld, and for good reason, since it offers the most balance between portability, price, and performance. It's a well-rounded machine that lets you play the latest PC games on the go with an intuitive UI and solid battery life for its specs. If this is your first PC gaming handheld rodeo, then the Steam Deck is easily the best choice. Meanwhile, the Asus ROG Ally is a great value in its own right due to it much lower price of entry that rivals the Steam Deck, its lightweight, gorgeous, and highly portable design, and its excellent specs and gaming performance. Either way, you really can't go wrong with either handheld.Governor Idris Attracts National Dignitaries To Kebbi
Manmohan Singh: India's 'Reforms' Man And Politician With A Difference
Support grows for Blake Lively over smear campaign claimEd Sheeran has apologised to Manchester United's new manager for interrupting him during a live Sky Sports interview. Ruben Amorim was speaking after United's 1-1 draw with Ipswich on Sunday when the singer walked up to hug analyst Jamie Redknapp. His intervention brought the interview to a halt before Redknapp told Sheeran to "come and say hello in a minute". Some people on social media said Sheeran had been "rude" and called for him to apologise. "Apologies if I offended Amorim yesterday, didn't actually realise he was being interviewed at the time, was popping to say hi and bye to Jamie," Sheeran wrote on Instagram. "Obvz feel a bit of a bellend but life goes on. Great game though, congrats on all involved." The game at Portman Road was Amorim's first since he left Portuguese club Sporting Lisbon to take over at United. Marcus Rashford scored after two minutes before Omari Hutchinson equalised for the home side. Read more from Sky News: Two Britons believed to be among missing after tourist boat sinks British soldiers injured in major traffic pile-up Cat wriggles through drainpipe with less-than-purrfect results Follow our channel and never miss an update. Sheeran, a lifelong Ipswich fan, holds a minority stake in the club. He was pictured celebrating after Hutchinson's goal. Be the first to get Breaking News Install the Sky News app for free Last week Ipswich CEO Mark Ashton said Sheeran helped the club sign a new player in the off-season by taking a Zoom call with him just before he went on stage with Taylor Swift.