Latest news with #CTX
Arabian Post
6 days ago
- Science
- Arabian Post
Mars Highlands Reveal Vast 15,000 km Ancient River Network
High‑resolution images from orbiting spacecraft have revealed an extraordinary network of over 15,000 km of fluvial sinuous ridges—also known as inverted channels—spanning Noachis Terra in Mars's southern highlands. This vast system, identified using data from Mars Reconnaissance Orbiter's HiRISE, CTX and MOLA instruments, indicates long‑lasting surface water activity shaped by precipitation roughly 3.7 billion years ago, reshaping scientific views of Mars's climate evolution. The ridges formed when river sediments cemented into resistant deposits, which later stood above the surrounding terrain after wind and erosion removed softer material. While previous research focused on valley networks, this study highlights inverted channels as compelling evidence of persistent, region‑wide water flow. Mapping uncovers meandering tributaries branching over hundreds of kilometres, with some avenues entering craters and breaching their rims—clear signs of river systems active long enough to carve into ancient impact landscapes. ADVERTISEMENT This finds new relevance in the Noachian‑Hesperian transition around 3.7 Ga—a geological era marked by a shift to a colder, drier Mars. The extensive fluvial systems preserved in Noachis Terra suggest sustained precipitation, rather than brief warming phases, supplied the water needed to maintain these rivers over a geologically meaningful period. The research, led by Adam Losekoot of the Open University and backed by the UK Space Agency, was unveiled at the Royal Astronomical Society's National Astronomy Meeting in Durham. Losekoot described Noachis Terra as a 'time‑capsule' recording ancient planetary processes, preserved for billions of years. These findings challenge earlier assumptions that equated Mars's early surface with a mostly cold and icy environment, punctuated by sporadic melting events. Instead, the new evidence supports a hypothesis of a warmer, wetter environment driven by substantial precipitation over extended periods. Noachis Terra had been relatively neglected by researchers focused on valley‑rich areas. The absence of traditional valley networks there previously led to underestimates of its water history. The focus on inverted channels opens fresh perspectives on how widespread surface water once was—even in terrains previously thought arid. This revived understanding of Mars's hydrological past connects with other findings that hint at subsurface water reserves. Among them, a recent international study reported a potential vast aquifer beneath Mars's south polar region. The new Noachis Terra data further supports the notion that early Mars had a robust water cycle, including precipitation and possibly rain‑fed riverine systems. Geologists also note that inverted channels have analogues on Earth, where cemented river sediments resist erosion and eventually form ridges that stand proud above eroded valleys. On Mars, such features appear most prominently in places like Miyamoto Crater and Juventae Chasma, but the scale of Noachis Terra's network is unprecedented. The implications for Mars's early environment are significant: a hydrologically active climate may have supported ecosystems or even nascent life. Though climate modelling has struggled to produce conditions that allow sustained liquid water, the physical evidence embedded in Noachis Terra's ridges demands revised scenarios. These might include episodic atmospheric thickening or greenhouse warming phases sufficient to sustain precipitation for extended times. Future research will likely probe whether similar inverted networks exist in other under‑studied highland regions and whether sediment composition points to seasonal cycles or sediment supply dynamics. Planned follow‑up with rover missions or crater‑site analysis may further evaluate if ancient lakes once sat behind these breached craters, and if mineral signatures—such as clay or sulphate layers—point to habitable or life‑friendly conditions.
Borneo Post
13-05-2025
- Health
- Borneo Post
Malaysian firm achieves breakthrough in stem cell therapy
KOTA KINABALU (May 13): A Malaysian company has been making breakthroughs in stem cell treatment following decades of research. According to Cytopeutics Sdn Bhd chief executive officer Prof Dr Chin Sze Piaw, the company is the first and only one in the country to obtain the Clinical Trial Exemption (CTX) licence for mesenchymal stem cells (MSC) for clinical use, issued by the Ministry of Health (MoH). In a recent programme undertaken at five MoH hospitals, one university hospital and one private hospital, they had been treating patients with cancer, specifically leukaemia, who underwent organ transplants but subsequently experienced recipient rejection. One particular child patient had graft-versus-host disease (GVHD), which occurs when the donor tissue reacts against the recipient's body. The child's body was breaking down, and he was bleeding from his nose, mouth, and under his skin. Dr Chin said the chances of survival in such cases are practically zero, as doctors would usually administer steroids, allowing the patients to survive for about three months before succumbing to cancer or infection. 'But our stem cells could overcome this. They come in like a mediator between the body and the transplant, and say, 'Hey, let's stop fighting and help each other.' 'And with our stem cells, it showed 100 percent survival for the patient within a year. Without the treatment, it was zero percent. 'MSC not only does not cause any cross-reactivity, it is actually used to treat such conditions,' he said at a special talk at a hotel here recently, organised by Maden Global Consultation (MGC) and Sunway MediSpa. Malaysia is one of six countries in the world that have approved the use of MSC for GVHD, and Cytopeutics is one of four companies to have received this approval. Dr Chin said they are also using stem cells to treat other significant diseases such as stroke, heart failure, diabetes, pre-diabetes and diabetes complications, autoimmune diseases like psoriasis, macular degeneration (blindness), and cancer. Among the treatments were cases involving patients on heart transplant waiting lists, where they demonstrated how they could eliminate scar tissue and create entirely new heart muscle for the patients. Aside from being the only Malaysian company that can manufacture stem cells for human clinical use safely, they are also the only one in the world to have ever received halal endorsement for the product. Cytopeutics has been working with the Department of Islamic Development (Jakim) since 2014 and has demonstrated for eight years that they are shariah-compliant, but they wanted to obtain the halal stamp. To do this, they had to show that their stem cells are produced in a halal manner. 'There are strict criteria to adhere to in the process of getting halal endorsement. 'The product has to be not just medically safe, but socially responsible and acceptable by all religions,' he explained. To this end, Dr Chin stressed to the public that not only are stem cells extracted from animals not halal, but they are also unsafe. Most people opt for animal-based stem cells because they are usually cheaper than safer alternatives. To get cheaper stem cells, the cells are grown using commercially available nutritional medium or animal serum/blood. Notably, animal stem cell therapy has been banned in almost all countries around the world. 'So, a lot of people would use blood or serum from cows or horses. This is not safe because the serum contains many viruses and antibodies,' he said. One way to assess the safety of stem cells is to ask the laboratory how they process their stem cells. If they refuse to answer, Dr Chin advised walking away. 'If you're injecting something into your body and it sounds too good to be true, it probably is,' he said. Cytopeutics uses MSCs for many of their treatments, which are approved by the regulatory bodies of the United States and China. MSC is akin to the O-negative red blood cell, which can be universally accepted by all blood types. The company started exploring stem cells obtained from umbilical cords about 26 years ago, despite being mocked as the structure is typically discarded. Twenty years later, it has proved to be one of the safest sources of stem cells for treatment. Dr Chin shared about a study involving three generations of families treated using MSCs, who underwent regular health checks. The infants' genes and chromosomes were inspected through full genomic sequencing, where each chromosome was unraveled to see how the genetic codes are arranged. The results of the study were very positive. 'We were the first and still the only ones to have completed a phase one human study on this, where we followed up with everybody for a minimum of five years. 'Now we are at 15 years, and throughout this time, nobody in the study has had heart attacks, diabetes, new infections or viruses, or cancer,' said Dr Chin. Also present were Universiti Malaysia Sabah (UMS) senior lecturer Dr Suhaini Sudi, UMS Faculty of Science and Natural Resources head of programme Dr Lucky Goh Poh Wah, Datuk Eliza Goh, MGC managing director Dona Chuan, MGC chief executive officer Alister Yong, and Sunway MediSpa general manager Dr Daniel Rejith.
Yahoo
24-02-2025
- Health
- Yahoo
FDA approves Mirum's Ctexli for cerebrotendinous xanthomatosis
Mirum Pharmaceuticals' Ctexli (chenodiol) has gained US Food and Drug Administration (FDA) approval for treating adults with cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease. It is the first drug approved by the US regulator for the condition. In June 2024, the company submitted a new drug application (NDA) for chenodiol to the US regulator to treat patients with CTX. Ctexli's efficacy was assessed in a 24-week, placebo-controlled, randomised, double-blind crossover withdrawal trial. Subjects treated with Ctexli, at a dose of 250 mg three times per day, showed a significant decrease in plasma cholestanol as well as urine 23S-pentol. The drug replaces deficiencies in bile acids, minimising abnormal cholesterol metabolite deposits. FDA Center for Drug Evaluation and Research, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine director Janet Maynard stated: 'The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis. 'CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments. Today's approval provides a safe and effective treatment option for CTX.' A liver toxicity warning for individuals with pre-existing liver conditions or bile duct abnormalities who are at an increased liver damage risk is included in the drug's prescribing information. Liver blood tests are recommended before commencing the treatment then annually thereafter, and as clinically indicated. Symptoms such as stomach pain, nausea and yellowing of the skin should prompt patients to discontinue the drug and consult their doctor. Common side effects of the drug include diarrhoea, abdominal pain and upper respiratory tract infections. Caused by a mutation in the cytochrome P450 family 27 subfamily A member 1 (CYP27A1) gene, CTX leads to a deficiency of an enzyme that is crucial for fat breakdown. This results in abnormal cholesterol metabolite deposition in several body parts, causing organ and tissue damage. In July 2023, the company expanded its bile acid product portfolio through a $445m acquisition deal with Travere Therapeutics. "FDA approves Mirum's Ctexli for cerebrotendinous xanthomatosis" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
24-02-2025
- Business
- Yahoo
Mirum's CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
CTEXLI is the first and only medication approved for the treatment of CTX in adults Approval based on Phase 3 RESTORE study results CTEXLI granted U.S. FDA Orphan Drug exclusivity for the treatment of CTX FOSTER CITY, Calif., February 24, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLI™ (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX). CTEXLI is the first and only treatment approved for this rare, progressive and debilitating disease. The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures. The primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant (p<0.0001). At the end of the randomized double-blind withdrawal period, there was a 20-fold difference between placebo and CTEXLI treated patients in urine 23S-pentol levels. In CTX, a deficiency of the bile acid chenodeoxycholic acid (CDCA) leads to a buildup of bile alcohols which precedes a toxic accumulation of cholestanol. Cholestanol is the key driver of symptomatic burden and disease progression, including irreversible neurologic dysfunction. Results from the RESTORE study demonstrated that treatment with CTEXLI not only improved urine bile alcohol levels but also serum cholestanol levels. Additionally, a greater proportion of patients receiving placebo required blinded rescue therapy, demonstrating the robustness of the effect. "The FDA's approval of CTEXLI is tremendous as it unlocks an opportunity to better identify and treat adult patients with CTX in the United States. Our hope is that patients are diagnosed sooner and have a chance to avoid some of the debilitating and lasting symptoms associated with CTX," said Chris Peetz, chief executive officer at Mirum. "We are grateful to the clinicians, patients, advocates, and families who participated in the research that led to this approval and who have continued to provide support to this community." "Cerebrotendinous xanthomatosis (CTX) is a rare disease that can present with early cataracts, tendon lipid deposits, and significant neurologic disease, and the latter may be prevented with earlier diagnosis and treatment," said Ernst J. Schaefer, MD, professor of medicine at Tufts University School of Medicine in Boston and chief medical officer and laboratory director at Boston Heart Diagnostics. "Treatment with CTEXLI has been shown to lower bile alcohols and cholestanol levels, reducing the progressive symptoms associated with CTX." "CTX is a devastating disease that is often diagnosed in early adulthood with progressive symptoms that can have a significant impact on a person's quality of life," said Jean Pickford, executive director, CTX Alliance. "We are thrilled that CTEXLI is now approved and hope that patients with this disease are diagnosed earlier and can avoid potentially irremediable disease progression and many of the debilitating symptoms associated with CTX." CTEXLI will be available through Mirum Access Plus (MAP), Mirum's patient support program. Patients currently on Mirum products or those with a prescription for CTEXLI can coordinate with MAP to receive fulfillment support by dialing (855) MRM-4YOU (855-676-4968) or visiting for more information. About Cerebrotendinous Xanthomatosis Cerebrotendinous xanthomatosis (CTX) is an autosomal, recessive, progressive genetic disorder resulting from a deficiency of a key enzyme in the bile acid synthesis pathway. CTX is characterized by fatty yellow nodules (xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body. As the clinical course progresses, irreversible neurological deterioration leads to premature death. CTX is a rare disease affecting one to two thousand people in the United States. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential benefits of CTEXLI for appropriate patients, and the real life CTEXLI treated patients' reduction of symptoms of CTX. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "will," "could," "can," "would," "potential," "hope," "opportunity," and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. A further description of risks and uncertainties can be found in Mirum's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at . View source version on Contacts Media Contact:Erin Murphymedia@ Investor Contact:Andrew McKibbenir@ Sign in to access your portfolio
Yahoo
19-02-2025
- Business
- Yahoo
Mirum Pharmaceuticals to Announce Fourth Quarter and Year-End 2024 Financial Results and Host Conference Call on February 26, 2025
FOSTER CITY, Calif., February 19, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will report fourth quarter and year-end 2024 financial results on Wednesday, February 26, 2025. Mirum will also host a conference call to discuss the fourth quarter and year-end 2024 financial results and recent corporate progress. Conference call details: Wednesday, February 26, 2025 4:30 p.m. ET / 1:30 p.m. PT Dial-in: U.S./Toll-Free: + 1 833 470 1428 International: + 1 404 975 4839 Passcode: 126145 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About Mirum Pharmaceuticals Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX). Mirum's late-stage pipeline includes investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. Lastly, Mirum is planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). View source version on Contacts Investor Contacts:Andrew McKibbenir@ Media Contact:Erin Murphymedia@ Sign in to access your portfolio
