Latest news with #X-ALD
Time of India
4 days ago
- Health
- Time of India
High-risk transplant saves 7-yr-old with rare disorder
New Delhi: A seven-year-old boy, diagnosed with X-linked Adrenoleukodystrophy (X-ALD) that is a rare and often fatal genetic disorder, has made a remarkable recovery after undergoing a high-risk stem cell transplant at Sir Ganga Ram Hospital. X-ALD is a genetic condition caused by the buildup of very long-chain fatty acids in the brain and spinal cord, leading to progressive neurological deterioration, sensory loss and death if left untreated. Early diagnosis and intervention are crucial for survival, doctors say. The boy has already lost his elder sibling to the same disease. Upon diagnosis by the hospital's genetics team, led by Dr Ratna Puri and Dr Sunita Bijarnia, he was referred to the pediatric hematology, oncology, and bone marrow transplant (BMT) unit. However, the transplant posed significant challenges as no fully matched donor was available within the family or in global registries. You Can Also Check: Delhi AQI | Weather in Delhi | Bank Holidays in Delhi | Public Holidays in Delhi Eventually, his father was chosen as a half-HLA matched donor. Serious complications followed the transplant. The child developed grade 4 graft-versus-host disease (GVHD), which caused widespread blistering and skin peeling. "There were moments when hope seemed to fade," said Dr Manas Kalra, a senior member of the treating team said, while adding that the boy's and his family's courage saw them through. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Seniors Are Ditching Finger Pricks with This Breakthrough Monitor Thelifeh Undo The child also developed a severe adenovirus infection that was unresponsive to conventional treatment. The infection significantly prolonged his hospital stay. As his new immune system began to develop, doctors were able to control the infection. Now, over 200 days post-transplant, the boy achieved full donor cell engraftment and is showing early signs of neurological recovery. He will remain under close follow-up in the coming years. While this child was not born of a consanguineous marriage, doctors stressed that such unions significantly increase the risk of inherited disorders like X-ALD. They emphasised the need for early genetic screening, improved public awareness, and stronger donor registry access. Doctors also highlighted the financial burden of such procedures and the need for wider support under rare disease programmes. "This success shows what is possible with timely diagnosis, advanced care, and community support," said Dr Kalra.
Yahoo
13-05-2025
- Business
- Yahoo
2 Monster Stocks in the Making to Buy Now and Hold for 10 Years
Viking Therapeutics has a promising pipeline candidate in the fast-growing therapeutic area of weight loss. Madrigal Pharmaceuticals markets the only approved therapy for a disease whose prevalence is growing. 10 stocks we like better than Viking Therapeutics › Investing in promising companies before their stock prices soar is an excellent recipe for earning life-changing returns. However, with hundreds of companies vying for investors' attention, separating the wheat from the chaff can be challenging. Recognizing those corporations with long-term market-beating potential -- especially before they are well-established -- isn't easy, but let's give it a shot anyway. Consider two mid-cap biotechs: Viking Therapeutics (NASDAQ: VKTX) and Madrigal Pharmaceuticals (NASDAQ: MDGL). These two drugmakers could grow in prominence in the next decade and deliver superior returns. Viking Therapeutics became famous in the biotech world last year after announcing positive phase 2 results for VK2735, an investigational GLP-1 weight loss candidate. The stock has not performed well since, which we can attribute to several factors. First, longtime investors took the opportunity to cash in on its big jump. Second, Viking hasn't had any clinical update as significant as that since. Third, like most other corporations, the drugmaker is suffering from marketwide issues. However, the bullish case for Viking is straightforward. The company could develop successful medicines in areas with unmet or growing needs. VK2735 has produced phase 2 data that rivals almost any other anti-obesity candidate outside those being developed by the leaders in the field, Eli Lilly and Novo Nordisk. Then there's VK2809, a promising candidate for metabolic dysfunction-associated steatohepatitis (MASH). Because obesity is a major risk factor for MASH, there's a significant need for therapies to address the condition. There is only one medicine approved by the U.S. Food and Drug Administration (FDA) for this disease, so there's room for others. VK2809, like VK2735, should make more progress this year. Elsewhere, Viking is developing VK0214 to treat X-linked adrenoleukodystrophy (X-ALD), a rare genetic nervous-system disorder. There are ways to manage X-ALD symptoms, but there is no disease-specific FDA-approved treatment. VK0214 has earned the orphan drug designation from the agency, which is reserved for therapies in development that have shown promising clinical evidence in treating rare diseases. So Viking's pipeline looks promising, and we haven't even mentioned the company's oral version of VK2735. True, the stock is somewhat risky, as are all clinical-stage biotech companies. However, if enough things go Viking's way, it could generate monster returns in the next decade as it earns approval for key products and starts generating strong revenue. In my view, initiating a small position in the stock is worth it. The only FDA-approved medicine for MASH, Rezdiffra, belongs to Madrigal Pharmaceuticals. It earned this honor early last year, although the medicine is under accelerated approval; that means it will have to prove efficacy once and for all in confirmatory studies, or it could be taken off the market. In the meantime, the drug is performing well. In the first quarter, its sales came in at $137.3 million, ahead of consensus analyst estimates; the medicine has made it a habit to top Wall Street's projections. It also speaks volumes about Madrigal. Plenty of drugmaking giants are looking to develop breakthrough therapies in this field. So far, only Madrigal has succeeded. Though another company will eventually challenge Madrigal, the future still looks bright. The biotech is already treating 17,000 patients with Rezdiffra, but that's still just about 5% of the 315,000 the company is targeting. Also, its crown jewel is approved for MASH patients with moderate to advanced liver fibrosis (scarring). Madrigal will seek label expansions in the most advanced form of scarring, cirrhosis. The biotech estimates that this indication could double its target market. And that's before we consider the fact that the number of people with MASH is increasing and, according to some estimates, will continue to do so well beyond the next 10 with potential competition on the way, Madrigal Pharmaceuticals has a massive addressable market. That, together with its first-mover advantage and potential clinical progress for Rezdiffra, could allow it to generate superior returns in the next 10 years. The company could be on the way to establishing itself as a biotech leader. Before you buy stock in Viking Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Viking Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $598,613!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $753,878!* Now, it's worth noting Stock Advisor's total average return is 922% — a market-crushing outperformance compared to 169% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 12, 2025 Prosper Junior Bakiny has positions in Eli Lilly, Novo Nordisk, and Viking Therapeutics. The Motley Fool recommends Novo Nordisk and Viking Therapeutics. The Motley Fool has a disclosure policy. 2 Monster Stocks in the Making to Buy Now and Hold for 10 Years was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
