Latest news with #Huntington's
Yahoo
5 days ago
- Health
- Yahoo
San Angelo family loses second daughter to Huntington's disease: Here's how to help
Just two years after losing their first daughter, a San Angelo family has lost their second daughter to Huntington's disease. On April 17, 2023, Khloe Salazar Gloria's battle with juvenile Huntington's disease came to an end at the age of 7. One year later in 2024, her parents, Natalie Salazar and Hector Gloria got the news that their younger daughter, Ariahella, had been diagnosed with the same rare and devastating illness. "Juvenile Huntington's Disease (JHD) is a rare genetic brain disorder that slowly takes away a child's ability to move, speak, eat, and think," Natalie said on a GoFundMe page. "There is no cure." Ariahella Kamille Gloria was reunited with her sister on June 29 at the age of 5. "I'm not sure how to live at this moment, I can only thank God for taking your pain away even in your last moments," Natalie said in a Facebook post. "I hope you know mama tried her best and I will forever feel sorry for your suffering and pain. I love you so much my little bull, my mermaid, my whole life." Natalie said she doesn't want the disease to define who Ari was. "Her constant laughter and smile that would fill the room will definitely be missed, but not forgotten as we will always continue to keep her memories alive," she said. A Facebook page called "Keeping UP with KHLOE & ALONG CAME ARI" was frequently updated about developments with the girls. It remains as a memorial page and will be updated with future fundraisers. Hector and Natalie thanked Hospice of San Angelo, her neurologist and medical team, her social worker Patrica and Candy, her hospice nurse. "We would also like to thank the community who have been following our story with both of our girls, we truly do appreciate all the support with much love and sadness," Natalie said. A GoFundMe titled "A Second Battle We Never Imagined – Supporting Ariahella" is available for donations. It is currently at $10,251 donations as of Thursday morning. "I know we have received such a large amount and that is all going to the funeral home and the plot at Lawnhaven," Natalie said in a message. "Any funds after that will be going to the headstone. We were blessed abundantly with Khloe." Natalie said there will be a brisket plate sale happening soon. Details will be posted on the Facebook page once finalized. A visitation for Ariahella will be held 6-8 p.m. Monday at Johnson's Funeral Home, 435 West Beauregard. A funeral service will be 10-11:30 a.m. Tuesday at The House Of Jireh, 1414 N. Chadbourne St. A graveside service be noon-1 p.m. at Johnson's Lawnhaven Memorial Gardens, 4989 FM Highway 1223. More: Live roaches on ice: Here's why the health department closed a San Angelo restaurant More: Farm to Fork: New grocery store opens in San Angelo selling homegrown foods This article originally appeared on San Angelo Standard-Times: San Angelo family loses daughters to Huntington's: Here's how to help
Yahoo
26-04-2025
- Health
- Yahoo
Family affected by Huntington's disease brings awareness to Siouxland
SIOUX CITY, Iowa (KCAU) — A family with a rare genetic disease is bringing awareness to Siouxland. Deb Conley has Huntington's Disease. According to the Huntington Study Group, four to five people out of 100,000 have the neurological disorder. 'Huntington's is kind of described as a combination of having Alzheimer's, Parkinson's and ALS all together in one,' Angie Conley, Deb's daughter-in-law, said. It is a genetic disorder, and Deb Conley's father and grandfather passed away from the disease. Norm Waitt Sr. YMCA opens new Early Learning and Youth Development Center 'It most commonly occurs to people in their thirties and forties, is when they start showing symptoms,' Angie said. 'Symptoms can be either kind of psychically and then they can have like some subtle personality changes. Other people show more cognitive executive skills type of changes. The career part of it, which is kind of your motor symptoms, you can have loss of coordination and movement, swallowing, talking, that type of thing.' There's a 50 percent chance a child of a parent with Huntington's will inherit the disease. Deb's son — Angie's husband — was recently diagnosed. 'He wasn't showing a lot of like the motor symptoms that spurred more recent,' Angie said. 'It was really difficult, but it also explained a lot for him. And it kind of put things into perspective for our family.' Despite the challenges the family faces, they say hope gets them through each day. 'It's a little more of a depressing type of disease, you know, having the disease if I didn't have my faith,' Deb said. The Conleys want to inform others about the disease and help fund research. On May 3, there will be walk for Huntington's disease awareness in Le Mars at Cleveland Park. 2nd annual Breakfast by the Bridge event raises awareness of youth homelessness 'We are doing a walk for hope for Huntington's disease,' Angie said. 'It's put on by the Huntington's Disease Society of America.' Research into Huntington's disease has improved how we understand the condition. 'It's changed over the years. when my grandpa was diagnosed, an autopsy was the only way you could find out. but now we've advanced so far in knowing what gene it is,' Deb said. Click this link if you want to know more about the Huntington's Disease Society of America's Iowa Statewide Team Hope Walk and want to register for the walk in Le Mars. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
11-04-2025
- Health
- Yahoo
Fundraiser in support of Huntington's Disease happening at Oak Mountain
PELHAM, Ala. (WIAT)– Huntington's Disease Society of America is hosting its 2025 Team Hope Walk on April 19 at Oak Mountain State Park. All proceeds from this event will go toward improving the lives of those affected with Huntington's Disease and their families. Huntington's Disease is a genetic disorder that causes the nerve cells in the brain to breakdown. Around 41,000 Americans are affected, while a further 200,000 are at risk. The children of those that suffer from Huntington's have a 50/50 chance of developing Huntington's. There is no cure. For registration and more information, click here. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Associated Press
17-03-2025
- Health
- Associated Press
Neurocrine Biosciences Presents New KINECT®-HD Data Showing Significant Reduction in Chorea Across Body Regions With INGREZZA® (valbenazine) Capsules
Data Reinforce the Clinically Meaningful Impact of INGREZZA in Adults with Huntington's Disease Chorea Findings Presented at the 2025 American Association of Neuroscience Nurses Neuroscience Advanced Practice Provider Symposium SAN DIEGO, March 17, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) presented data from the KINECT®-HD study showcasing significant improvements in chorea across body regions with once-daily INGREZZA® (valbenazine) capsules in adults with Huntington's disease. The post-hoc analysis was presented at the Neuroscience Advanced Practice Provider Symposium hosted by the American Association of Neuroscience Nurses in New Orleans. 'This analysis reinforces the primary study findings, showing the consistent benefit of INGREZZA in reducing the involuntary movements of chorea that can affect any part of the body,' said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. 'Notably, the most significant improvements were seen in the arms and legs, which were the most severely affected at baseline. These results are meaningful because they show how INGREZZA alleviates chorea symptoms and therefore helps people with Huntington's disease in their everyday lives.' In the 12-week, Phase 3 KINECT-HD clinical trial, once-daily INGREZZA demonstrated a significant reduction in chorea severity, as measured by the Unified Huntington's Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score, which measures chorea in seven body regions (face, buccal-oral-lingual region, trunk, right and left upper extremities, right and left lower extremities). This post-hoc analysis was conducted to assess the effect of INGREZZA on chorea by individual body region. In the KINECT-HD study, patients were randomized 1:1 to receive INGREZZA (n=64) at doses of 40 mg, 60 mg or 80 mg, or placebo (n=61). TMC scores at baseline were similar between treatment groups. Key findings from the post-hoc analysis include: More participants had potentially meaningful chorea improvements (a TMC score shift from ≥2 at screening/baseline to ≤1 at maintenance) with INGREZZA versus placebo for all affected body regions. Statistically significant chorea improvements were observed for the upper and lower extremities (P<0.05 for each) – body regions with the highest chorea severity seen at baseline. Average TMC body region scores improved at maintenance, with numerically greater improvements for all TMC items with INGREZZA versus placebo. About Chorea Associated with Huntington's Disease Huntington's disease (HD) is a hereditary progressive neurodegenerative disorder in which the loss of certain neurons within the brain causes motor, cognitive and psychiatric symptoms. Symptoms generally appear between the ages of 30 and 50 years and worsen over a 10- to 25-year period. Most people with HD experience chorea, an abnormal involuntary movement disorder, characterized by irregular and unpredictable movements. Chorea can affect various body parts and interfere with motor coordination, gait, swallowing and speech. HD is estimated to affect approximately 41,000 adults in the U.S., with more than 200,000 at risk of inheriting the disease. About KINECT®-HD KINECT®-HD was a Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy of valbenazine as a once-daily treatment to reduce chorea associated with Huntington's disease (HD) and evaluate the safety and tolerability of valbenazine in patients with HD. The study enrolled 128 adults 18 to 75 years of age who were diagnosed with motor-manifest HD and who had sufficient chorea symptoms to meet study protocol criteria. KINECT-HD used the Unified Huntington's Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score as the primary efficacy endpoint. The secondary endpoints included Clinical Global Impression of Change (CGI-C) response status and Patient Global Impression of Change (PGI-C) response status for valbenazine treatment. Treatment with valbenazine resulted in a placebo-adjusted mean reduction in the TMC score of 3.2 units (P<0.0001), indicating a substantial improvement in chorea. Secondary endpoints of CGI-C response status and PGI-C response status were also statistically significant and supported the improvements in TMC score that were seen over the 12-week study period. Treatment-emergent adverse events in this study were generally consistent with the known safety profile of valbenazine. The most common adverse reactions in patients with HD included somnolence and sedation, urticaria, rash and insomnia. View the complete study results from the Phase 3 KINECT-HD study published in The Lancet Neurology online edition. For more information on the KINECT-HD study, please visit About INGREZZA® (valbenazine) Capsules and INGREZZA® SPRINKLE (valbenazine) Capsules INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved by the U.S. Food and Drug Administration for the treatment of adults with tardive dyskinesia and the treatment of chorea associated with Huntington's disease (HD). Only INGREZZA offers a therapeutic dose from day one with no required titration. INGREZZA, developed by Neurocrine Biosciences, selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic or muscarinic receptors. While the specific way INGREZZA works to treat TD and HD chorea is not fully understood, INGREZZA is unique in that it selectively and specifically targets VMAT2 to inhibit the release of dopamine, a chemical in the brain that helps control movement. INGREZZA is believed to reduce extra dopamine signaling, which may lead to fewer uncontrollable movements. INGREZZA is proven across the widest range of patients. It is always one capsule, once daily and can be taken together with most stable mental health regimens such as antipsychotics or antidepressants. Only INGREZZA offers the benefit of a sprinkle formulation, INGREZZA® SPRINKLE, for those who experience dysphagia, have difficulty swallowing or prefer not to swallow a pill. INGREZZA and INGREZZA SPRINKLE dosages approved for use are 40 mg, 60 mg and 80 mg capsules. Important Information Approved Uses INGREZZA® (valbenazine) capsules or INGREZZA® SPRINKLE (valbenazine) capsules are prescription medicines used to treat adults with: movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia). involuntary movements (chorea) of Huntington's disease. INGREZZA or INGREZZA SPRINKLE do not cure the cause of involuntary movements, and do not treat other symptoms of Huntington's disease, such as problems with thinking or emotions. It is not known if INGREZZA or INGREZZA SPRINKLE is safe and effective in children. IMPORTANT SAFETY INFORMATION INGREZZA or INGREZZA SPRINKLE can cause serious side effects in people with Huntington's disease, including: depression, suicidal thoughts, or suicidal actions. Tell your healthcare provider before you start taking INGREZZA or INGREZZA SPRINKLE if you have Huntington's disease and are depressed (have untreated depression or depression that is not well controlled by medicine) or have suicidal thoughts. Pay close attention to any changes, especially sudden changes, in mood, behaviors, thoughts, or feelings. This is especially important when INGREZZA or INGREZZA SPRINKLE is started and when the dose is changed. Call your healthcare provider right away if you become depressed, have unusual changes in mood or behavior, or have thoughts of hurting yourself. Do not take INGREZZA or INGREZZA SPRINKLE if you: are allergic to valbenazine, or any of the ingredients in INGREZZA or INGREZZA SPRINKLE. INGREZZA or INGREZZA SPRINKLE can cause serious side effects, including: Allergic reactions. Allergic reactions, including an allergic reaction that causes sudden swelling called angioedema can happen after taking the first dose or after many doses of INGREZZA or INGREZZA SPRINKLE. Signs and symptoms of allergic reactions and angioedema include: trouble breathing or shortness of breath, swelling of your face, lips, eyelids, tongue, or throat, or other areas of your skin, trouble with swallowing, or rash, including raised, itchy red areas on your skin (hives). Swelling in the throat can be life-threatening and can lead to death. Stop taking INGREZZA or INGREZZA SPRINKLE and go to the nearest emergency room right away if you develop these signs and symptoms of allergic reactions and angioedema. Sleepiness and tiredness that could cause slow reaction times (somnolence and sedation). Do not drive a car or operate dangerous machinery until you know how INGREZZA or INGREZZA SPRINKLE affects you. Drinking alcohol and taking other medicines may also cause sleepiness during treatment with INGREZZA or INGREZZA SPRINKLE. Heart rhythm problems (QT prolongation). INGREZZA or INGREZZA SPRINKLE may cause a heart rhythm problem known as QT prolongation. You have a higher chance of getting QT prolongation if you also take certain other medicines during treatment with INGREZZA or INGREZZA SPRINKLE. Tell your healthcare provider right away if you develop any signs or symptoms of QT prolongation, including: fast, slow, or irregular heartbeat (heart palpitations), shortness of breath, dizziness or lightheadedness, or fainting or feeling like you are going to faint. Neuroleptic Malignant Syndrome (NMS). NMS is a serious condition that can lead to death. Call a healthcare provider right away or go to the nearest emergency room if you develop these symptoms and they do not have another obvious cause: high fever, stiff muscles, problems thinking, irregular pulse or blood pressure, increased sweating, or very fast or uneven heartbeat. Parkinson-like symptoms. Symptoms include: body stiffness, drooling, trouble moving or walking, trouble keeping your balance, shaking (tremors), or falls. Before taking INGREZZA or INGREZZA SPRINKLE, tell your healthcare provider about all of your medical conditions including if you: have liver or heart problems, 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. Make sure you tell all of your healthcare providers that you are taking INGREZZA or INGREZZA SPRINKLE. Taking INGREZZA or INGREZZA SPRINKLE with certain other medicines may cause serious side effects. Especially tell your healthcare provider if you: take digoxin or take or have taken a monoamine oxidase inhibitor (MAOI) medicine. You should not take INGREZZA or INGREZZA SPRINKLE if you are taking, or have stopped taking, a MAOI within the last 14 days. The most common side effect of INGREZZA or INGREZZA SPRINKLE in people with tardive dyskinesia are sleepiness and tiredness. The most common side effects of INGREZZA or INGREZZA SPRINKLE in people with chorea associated with Huntington's disease include sleepiness and tiredness, raised itchy red areas on your skin (hives), rash, and trouble getting to sleep or staying asleep. These are not all of the possible side effects of INGREZZA or INGREZZA SPRINKLE. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at or call 1-800-FDA-1088. Dosage Forms and Strengths: INGREZZA and INGREZZA SPRINKLE are available in 40 mg, 60 mg, and 80 mg capsules. Please see full Prescribing Information, including Boxed Warning, and Medication Guide. 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. 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 LinkedIn, X and Facebook. (*in collaboration with AbbVie) The NEUROCRINE BIOSCIENCES Logo, NEUROCRINE, YOU DESERVE BRAVE SCIENCE, KINECT and INGREZZA are registered 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 INGREZZA for the treatment of chorea associated with Huntington's disease (HD) and the value INGREZZA may bring to patients with chorea associated with HD. 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 INGREZZA for the treatment of chorea associated with HD; whether INGREZZA receives adequate reimbursement from third-party payors; the degree and pace of market uptake of INGREZZA for the treatment of chorea associated with HD; risks and uncertainties relating to competitive products and technological changes that may limit demand for INGREZZA for the treatment of chorea associated with HD; risks associated with the Company's dependence on third parties for development and manufacturing activities related to INGREZZA for the treatment of chorea associated with HD, and the ability of the Company to manage these third parties; risks that additional regulatory submissions for INGREZZA for the treatment of chorea associated with HD 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 INGREZZA for the treatment of chorea associated with HD; risks that post-approval INGREZZA commitments or requirements may be delayed; risks that INGREZZA for the treatment of chorea associated with HD 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; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's annual report on Form 10-K for the year ended December 31, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law. © 2025 Neurocrine Biosciences, Inc. All Rights Reserved. CP-VBZ-US- 0049 03/2025
The Guardian
14-02-2025
- Politics
- The Guardian
The concerted attack on assisted dying won't stop the public supporting this bill
Don't be taken in. Campaigners try to create an impression that the herd is moving their way, hoping to set off a stampede. ''Chaotic' assisted dying bill at risk over High Court U-turn', goes one headline this week. But as the assisted dying bill makes its slow way through detailed scrutiny in Kim Leadbeater's committee, it's not happening. Only in the pages of the Tory press are MPs stampeding: actual MPs seem to be moving little, and if anything in favour, according to the campaign group Dignity in Dying. The vote at second reading was won by 55. Leadbeater's amendment to replace a high court judge's approval for an assisted death with a panel of three professionals – a psychiatrist, social worker and lawyer – set off an avalanche of claims that this was tipping the vote against her. Here's the Mail's headline: 'Social workers to sit on 'death panels' in Labour MP's watered-down assisted dying bill'. Social workers! According to the Mail's story, MPs who had previously backed the bill 'said they were now thinking again amid an angry backlash'. The Times quoted 10 Labour MPs signing a protest, without mentioning that these 10 MPs were always against assisted dying. The Times has been relentless. According to one of its reports, medical experts have warned that amendments to the bill could be a 'slippery slope' – the experts being, of course, those who were always anti-assisted dying. Leadbeater's amendment is due to practical evidence from the likes of the former chief justice Jonathan Sumption that high court judges in an overwhelmed court system couldn't take this on. Nicholas Mostyn, a former high court judge, gave evidence that the bill 'would not fly at all' without 'enough judges to go around'. The justice ministry requested this change, though the justice secretary, Shabana Mahmood, opposed it. The former president of the supreme court Brenda Hale gets much coverage for suggesting a district or circuit judge could take this on, but as a prominent supporter of assisted dying, she told the Financial Times that she didn't think 'the proposed panel is necessarily a bad thing'. Rather less coverage goes to her support for an amendment, backed by Humanists UK (of which I am vice-president), that extends eligibility for assisted dying to 12 months before death, not six months, to allow for those with neurological conditions such as motor neurone disease or Huntington's. Amendment 234, which is highly unlikely to pass, lays the ground for a future extension. Most people travelling to die at Dignitas in Switzerland have longer than six months to live. Polling for Humanists UK shows a majority of voters support there being no time limits for those with degenerative diseases such as multiple sclerosis, by 73% to 9% against. This is just one of more than 350 proposed amendments to the bill. Many of these seem intended to gum up proceedings, so when the committee ends by mid-April opponents can protest that too many amendments went undebated. All kinds of angles and wrangles are in play. How about this indignation from the Telegraph: 'Depressed people who are terminally ill could be eligible for assisted dying.' Some, who are dying in pain and seek a quicker death, may indeed be depressed. But if that disqualifies them, it's a catch-22. Should only the cheerful be qualified to end their lives? The well-financed but opaque campaign Care Not Killing has put out a tidal wave of online messages to postcode-targeted voters featuring a picture of their local MP. They feature the following message: 'Breaking News. MP 'watering down' assisted dying protections. Your MP must reconsider their position.' Typical is one sent to abstainer Chris Bryant's voters, which cost the campaign in the region of £500 to be seen up to 15,000 times on a screen. Will MPs take fright? I'd imagine not: the polls for years have been strongly in favour of assisted dying. YouGov's tracker holds steady, with 68% of the public supporting assisted dying. Others are even higher. The Labour MP Lewis Atkinson, a Leadbeater supporter who sits on the bill committee, has been subjected to such ads, along with a prolific religiously organised write-in. Opponents on the committee, he told me, are trying to kill the bill with delays: the committee spent 10 hours over the first two days of this week discussing one clause. When some near-identical amendment was refused, Danny Kruger, a committee member and lead opponent of assisted dying, tweeted out that the committee has refused to exclude people with impaired judgment or those 'unduly influenced' by others. Atkinson said this was nonsense, explaining that an amendment using the word 'manipulated' might not be selected over another near-identical one using the proper legal term 'coerced'. . But they can claim the bill's proponents rejected the idea of manipulation. He fears they will run the committee out of time, and then claim that the debate was stifled. The argument made by the UK's highest-ranking Catholic bishop, Cardinal Vincent Nichols, who claimed that MPs spent 'more than a hundred times longer debating foxhunting than assisted dying', will certainly not be the case. The religious have a considerable army behind them, but mostly they are not as honest about their God-driven reasons for opposing the bill as the single campaigner I spotted recently in Westminster with a placard reading 'Suicide is self-murder. Don't go to hell.' The point of a bill committee is to take screeds of evidence in order to improve it. But you can expect any change to the bill to be greeted by sham indignation from those who have always been opposed to it. The last word goes to the Express, the bill's unexpected sole champion in the rightwing media. Headlined 'Plotters against assisted dying bill must not win', it concludes 'the wreckers within Westminster should know that millions of people will be outraged if this vital reform is delayed. We will not give up this fight.' It only takes 28 MPs to change their mind for the bill to fail. Polly Toynbee is a Guardian columnist
