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Researchers suggest treatments for rare diseases could reduce burden of kidney disease

Rare kidney diseases are a group of conditions which account for over 5% of people living with chronic kidney disease A new study led by University College London (UCL) and the UK Kidney Association has suggested that treatments for rare diseases could significantly reduce the burden of kidney disease for patients and the NHS. Published in The Lancet in alignment with World Kidney Day (14 March 2024), the study draws on the largest kidney disease dataset ever created, the UK’s National Registry of Rare Kidney Disease (RaDaR). Affecting over seven million adults, according to Kidney Research UK, chronic kidney disease (CKD) is characterised by the gradual loss of kidney function over a period of months to years. Rare kidney diseases such as cystinosis and Fabry disease are a group of conditions that affect less than one in 2,000 people, accounting for over 5% of people with CKD. Researchers charted the ...
- Source: drugdu
- 79
- March 22, 2024
New study reveals neurological conditions as leading cause of ill health worldwide

In 2021, neurological conditions, including Alzheimer’s disease, affected approximately 3.4 billion people globally In a new study funded by the Bill & Melinda Gates Foundation, results published in the Lancet Neurology have determined that neurological conditions are currently the leading cause of ill health worldwide. Results showed that the biggest contributors to neurological health loss globally were stroke, neonatal encephalopathy, migraine, Alzheimer’s disease and other dementias, and diabetic neuropathy. Affecting 3.4 billion people globally in 2021, neurological conditions are any conditions that affect the brain, spinal cord and nerves, which can affect anyone at any age. The study revealed that the number of people globally living with or dying from neurological conditions has drastically risen in the past three decades, partially due to ageing societies. Worldwide, the analysis suggests that the total amount of disability, illness and premature death caused by neurological conditions increased by 18% between 1990 and 2021. ...
- Source: drugdu
- 128
- March 22, 2024
Everest Medicines Announces Singapore Health Sciences Authority Approves NEFEGAN® for the Treatment of Primary IgA Nephropathy in Adult Patients

Shanghai, China – March. 20, 2024 — Everest Medicines (HKEX 1952.HK, “Everest”, or the “Company”), a biopharmaceutical company focused on the discovery, development, manufacturing and commercialization of innovative medicines and vaccines, announced today that the Singapore Health Sciences Authority (HSA) has approved NEFEGAN® for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk of disease progression. NEFEGAN®, known in other Everest territories as Nefecon®, was the first ever treatment for IgAN fully approved by the U.S. Food and Drug Administration, and Singapore marks the third region in Everest territories that received New Drug Application (NDA) approval after Macao and mainland China. “The NDA approval in Singapore marks an important event for IgAN patients in the region as we continue to expand patient access to Nefecon® throughout Asia, an area of high IgAN disease prevalence,” said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. “Following approval of ...
- Source: drugdu
- 94
- March 22, 2024
Accelerating stakeholder collaboration to enhance the clinical trials environment in the EU

The Accelerating Clinical Trials in the EU (ACT EU) initiative has established a multi-stakeholder platform (MSP) aimed at improving the environment for clinical trials across the European Union (EU). ACT EU is a collaboration between EMA, the Heads of Medicines Agencies (HMA) and the European Commission (EC), which seeks to transform how clinical trials are initiated, designed, and run. During the first meeting of the MSP advisory group that officially kicks-off the platform, María Jesús Lamas, director of the Spanish agency of medicines and medical products, who is regulatory co-chair of the group, said: “The establishment of the multi-stakeholder platform is an important milestone for ACT EU. Our collaborative efforts will drive progress towards more streamlined and efficient clinical trials and allow patients and citizens to benefit from clinical research in Europe.” The launch of the MSP fosters collaboration and promotes open dialogue around the challenges and opportunities represented by ...
- Source: drugdu
- 115
- March 22, 2024
Blood Test Identifies Individuals at Highest Risk of Dying From Heart Failure

Heart failure is a serious condition where the heart is unable to pump blood effectively throughout the body, leading to frequent hospital stays and a decline in quality of life. A protein known as neuropeptide Y (NPY) is produced by nerves in the heart under severe stress. NPY can cause dangerous heart rhythms and narrow the heart’s smallest blood vessels, forcing the heart to work harder and narrowing the arteries that supply blood to the heart. Now, a new study has found that measuring NPY levels could predict the progression of heart failure, paving the way for a blood test to assist in managing heart failure treatment within the next five years. The collaborative study by researchers at the University of Oxford (Oxford, UK) and University of Glasgow (Scotland, UK) found that patients with the highest NPY levels had a 50% greater chance of dying from heart complications within three ...
- Source: drugdu
- 97
- March 21, 2024
Improved Microneedle Technology Speeds Up Extraction of Sample Interstitial Fluid for Disease Diagnosis

Interstitial fluid has many similarities with blood, and its secrets are still being uncovered. A microneedle offers a minimally invasive method to sample this fluid directly under the skin. This tool allows for real-time and continuous monitoring of biomarkers circulating in the body. Despite their small size, just two to three times the width of a human hair and around a millimeter in length, microneedles can make a significant difference in early infection diagnosis and personal health monitoring. Now, researchers have developed improved microneedle technology that enhances the extraction of interstitial fluid by collecting more sample quantities in less time. Sandia National Laboratories (Albuquerque, NM, USA) is leading the way in microneedle research through collaboration with different partners to enhance this technology. Sandia has achieved a breakthrough in interstitial extraction, moving from using multiple needle arrays to a single microneedle technique that collects enough fluid for analysis in just about ...
- Source: drugdu
- 85
- March 21, 2024
AstraZeneca Joins Radiopharma Deals Spree With $2B Fusion Acquisition

AstraZeneca’s Fusion Pharmaceuticals acquisition is the latest in a wave of M&A activity that includes recent deals by Eli Lilly and Bristol Myers Squibb. The lead Fusion program is in mid-stage clinical development for treating advanced prostate cancer. By FRANK VINLUANAstraZeneca is following in the footsteps of some of its big pharmaceutical company peers, plunking down $2 billion to acquire its radiopharmaceuticals partner Fusion Pharmaceuticals. More than adding a pipeline of targeted radiation therapies, AstraZeneca gains the crucial supply chain and manufacturing infrastructure to support them. The sum is an upfront payment. According to the deal terms announced Tuesday, AstraZeneca will pay $21 cash for each Fusion share, representing a 97% premium to Fusion’s closing stock price on Monday. Shareholders of the radiopharmaceutical company could get even more. The deal includes a contingent value right that will pay an additional $3 per share upon achievement of a regulatory milestone. That ...
- Source: drugdu
- 138
- March 21, 2024
FDA Grants Accelerated Approval to Takeda’s Iclusig for Ph-Positive Acute Lymphoblastic Leukemia

Davy James Label expansion approval for Iclusig (ponatinib) addresses adult patients with newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia. The FDA has granted accelerated approval to Takeda’s supplemental New Drug Application for Iclusig (ponatinib) plus chemotherapy to treat adults with newly diagnosed Philadelphia chromosome (Ph)–positive acute lymphoblastic leukemia (ALL).1,2 Iclusig is the only pan-mutational, third-generation tyrosine kinase inhibitor (TKI) that targets BCR::ABL1, an abnormal tyrosine kinase expressed in Ph-positive ALL and chronic myeloid leukemia (CML). Iclusig can also treat all known single, treatment-resistant mutations, including T315I. “This label expansion for Iclusig is an incredibly exciting milestone, allowing US adult patients with newly diagnosed Ph-positive ALL to have an approved, targeted treatment option in the frontline,” Awny Farajallah, MD, chief medical officer, oncology at Takeda, said in a press release. “We are thrilled that the FDA has recognized the potential of Iclusig to fill a large gap in care for these ...
- Source: drugdu
- 163
- March 21, 2024
BIO-THERA Signs License and Commercialization Agreement with SteinCares for Two Investigational Biosimilars

BIO-THERA Biopharmaceutical Co., Ltd (SSE: 688177), a global science-based and innovative biopharmaceutical company based in Guangzhou, China, hereinafter referred to as “BIO-THERA” or the “Company”, today announced that it has signed license and commercialization agreements with SteinCares for two investigational biosimilars. The Company today announced that it has entered into a license and commercialization agreement with SteinCares for two investigational biosimilars. Under the terms of the agreements, SteinCares will have exclusive distribution and marketing rights for the two biosimilars in Brazil and the rest of Latin America. Under the terms of the agreements, BIO-THERA will be responsible for the development, manufacturing, commercial supply and filing of marketing applications with the U.S. FDA and the European EMA for the two biosimilars, and SteinCares will be responsible for the filing of marketing applications for the two biosimilars in Brazil and the rest of Latin America, and for their commercialization. SteinCares is familiar ...
- Source: drugdu
- 169
- March 21, 2024
ALS Association partners with myTomorrows to improve clinical trial accessibility

The fatal motor neurone disease currently does not have a cure available to treat patients The ALS Association and myTomorrows have partnered to improve the accessibility of future clinical trials for patients living with amyotrophic lateral sclerosis (ALS). The aim of the partnership is to ensure that patients living with the condition and physicians are up-to-date with information about pre-approval treatment options and clinical trials. ALS is a fatal motor neurone disease that is characterised by the progressive degeneration of nerve cells in the spinal cord and brain, affecting the voluntary control of arms and legs. Usually fatal within five years with no cure available, searching for clinical trials for ALS patients is challenging due to its day-to-day implications and rapid escalation. The US-based ALS Association and myTomorrows aim to tackle these challenges and make it easier for patients and their families to participate in clinical trials. The ALS Association ...
- Source: drugdu
- 164
- March 21, 2024

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