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Volume 1
Issue 2 JULY– DECEMBER 2025
Volume 1 (Issue 2) JULY– DECEMBER 2025 Research Articles
Real-life Effects of Enzyme Replacement Therapy in Adults with Late Onset Pompe Disease: Multicentric Retrospective Study
Vol.1(2); Pages:1-7. Published on July-2025
Abstract
Pompes disease is an infrequent lysosomal storage disease that is brought on by a deficiency in acid alphaglucosidase, resulting in muscle weakness that develops gradually and respiratory impairment. This retrospective multicenter study aims at assessing the clinical effects of enzyme replacement therapy (ERT) in 52 adult patients with late-onset Pompe disease (LOPD) during the 3-year treatment. It involved an analysis of data of five European specialty centers, with emphasis on the changes of forced vital capacity (FVC), 6-minute walk distance (6MWD), and adverse events. ERT was linked to the statistically significant mean changes of 9 percent in FVC and 14 percent in 6MWD (p < 0.05). All but one adverse event was a mild infusion reaction and no patient discontinued. These data confirm the long-term effectiveness and safety of ERT to enhance the respiratory function and mobility in adult LOPD patients, which provides the persistence of access to specialized pharmacotherapy as an approach to managing rare diseases.
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Delta Phase II Open-Label Trial Enzyme Replacement Therapy with Pegylated Alpha-Glucosidase in Late-Onset Pompes Disease
Vol.1(2); Pages:8-15. Published on July-2025
Abstract
Pompes disease is a general infantile progressive autosomal recessive disease due to the deficit of acid alphaglucosidase (GAA), which causes a lysosomal storage condition with glycogen blocking and weakened and weakened muscles and respiratory deterioration. This was a Phase II open-label study to examine safety and effectiveness of pegylated recombinant alpha-glucosidase enzyme in 38 adults who had late-onset Pompe disease. The duration of intravenous infusion comprised 12 months through weekly infusions in patients. The primary clinical outcomes measures were the six-minute walk (6MWT), forced vital capacity (FVC) and safety. The outcome indicated a 38 meter (average) increase in 6MWT and stabilization of FVC in 71 and 64 percent of the patients respectively. Majority of the adverse events were minor and mild-moderate infusion-related events. In contrast to standard therapy, plasma stability was greater, and immunogenicity was diminished in the pegylated formulation, suggesting the potential of long-term therapy.
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A Multinational Observational Study Real-World Treatment Patterns of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
Vol.1(2); Pages:16-24. Published on July-2025
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal hematopoietic stem cell disease characterized by uncontrolled activation of the activation of the complement systems, intravascular hemolysis, and the threat of thrombosis. The present multinational, observational study assessed the use of eculizumab in the real world of 172 patients of 14 European centers during 24 months. The important outcomes were the transfusion independence, thromboembolism, breakthrough hemolysis, and patient-reported outcome. Sixty-one percent of patrons became transfusion-independent at 12 months, and the rate of thromboembolism markedly reduced relative to that before treatment (p < 0.01). There was breakthrough hemolysis in 18% which was mainly caused by suboptimal dosing intervals. There was a strong improvement in patient-reported fatigue and disease burden. Such results show that eculizumab has convincing clinical utility in daily practice, with the challenge of dose individualization and other long-acting complement inhibitors.
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Two-year follow-up of gene Therapy in Type I spinal muscular atrophy with aav9-mediated SMN1 Delivery Results
Vol.1(2); Pages:25-32. Published on August-2025
Abstract
Spinal muscular atrophy type I (SMA-I) is a critical autosomal disease that is recessive in nature and is as a result of homozygous deletion or mutation of the SMN1 gene. This prospective longitudinal study assessed 24 children with a single intravenous administration of adeno-associated virus serotype 9 (AAV9) gene therapy intervention to deliver SMN1 in children. Patients were followed up to 24 months with 91 percent survival and 54 percent reached independent sitting- a milestone not often observed in untreated SMA-I. Complications were mostly transaminitis that was successfully treated by corticosteroid prophylaxis. None of the oncogenesis or systemic toxicity in relation to vectors was detected. Survival and visceral improvement was very high in treated infants than natural history. These results lend credence to AAV9-SMN1 gene therapy, which may be transformative in SMA-I, but longer follow-up is needed.
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Meta-analysis review and reviewing mTOR inhibitors in the case of the tuberous sclerosis complex-related tumors
Vol.1(2); Pages:33-41. Published on August-2025
Abstract
Tuberous sclerosis complex (TSC) is a rare genetically inherited condition that is presented by tumors (hamartomatous) of various organs. The mechanism of action of rapamycin to TSC is majorly by acting through its mechanistic target of rapamycin (mTOR) pathway, hence inhibition of mTOR will act as a selective therapeutic medication. This meta-analysis and systematic review included 11 clinical trials (4 randomized controlled trials and 7 observational cohort studies) including 812 patients who received the everolimus, or the sirolimus. Combined outcomes showed 48% tumor decrease in patients undergoing treatment, and 9%, in controls. The subgroup analysis was advantageous in subependymal giant cell astrocytomas and renal angiomyolipomas. Marginal reductions were noted on the number of seizures and the general survival. Side effects could be dealt with re. Dose modifications. These observations advocate mTOR inhibitors in approaching TSC-related tumors and modifying the disease.
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Issue 1 JANUARY- JUNE 2025
Volume 1(Issue 1) JANUARY- JUNE 2025 Research Articles
Intelligent Clinical Copilot for Comprehensive Medical Risk Assessment
Vol.1(1); Pages:1-8. Published on May-2025
Abstract
Because healthcare is always changing, pharmacy programs now need to develop students who are expert, fast LLMs have recently shown impressive results on mock medical exams, but they struggle when existing models need to make clinical decisions that involve thinking logically about uncertainty. We develop RiskAgent which works alone as an autonomous copilot, addressing medical risk prediction by using more than 387 ready clinical tools. As an alternative to solely fine-tuning or making prompts, RiskAgent uses well-established tools to make sure the outcomes it creates are accurate, clear to interpret and reliable. We build MedRisk, the first benchmark designed for medical risk prediction, by gathering 12,352 cases representing 154 diseases, 86 symptoms, 50 specialties and 24 organ systems. The 8B-parameter version of RiskAgent reaches 76.33% accuracy which is much greater than that of the leading commercial LLMs GPT-4.5 and o1 and more than two times that of GPT4o. Significantly, the model demonstrates strong performance in cases where data is low and the disease specialty is rare or Cancer. Using a modular approach and trusted tools from medicine, RiskAgent ensures its deployment is waste-free, lowers costs and keeps patient privacy in mind. Open-source models from S7 (7B parameters) to S1 (1B parameters) are included in the system, together with code and datasets.
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Comparing the 2015 ACPE Accreditation Framework with the 2018 Chinese Clinical Pharmacy Education Standards: An Analysis
Vol.1(1); Pages:9-17. Published on May-2025
Abstract
The study carries out a comprehensive comparison between the 2018 National Accreditation Standard for Teaching Quality of Clinical Pharmacy in China and the 2015 Accreditation Standards and Guidelines of the ACPE in the United States. Because China is quickly developing its healthcare system and now needs reliable, high-quality pharmaceutical training on par with international standards, this comparison has become important. The paper compares the main aspects, structure of the curriculum, governance, support for students, teacher qualifications and evaluation methods of both systems. As seen by the study, although China’s 2018 accreditation standard is a key achievement for developing clinical pharmacy education, significant differences still exist. These challenges consist of low attention to curriculum changes, poorly specified roles for administrators and a shortage of well-defined methods to assess both student progress and the success of programs. Furthermore, language and rules in China serve to lock its education system out of the global and professional world. The final section of the study recommends steps for bringing China’s accreditation up to the global level expected for Pharm.D. programs. Among these, the initiative involves broadening the science curriculum, establishing business and leadership plans, supporting quality in teaching and making closer connections to the outcomes aimed by ACPE.
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Improving Healthcare for Rare Genetic Conditions in China: A Critical Call
Vol.1(1); Pages:18-26. Published on May-2025
Abstract
Since rare genetic diseases affect only a few people, together they influence many and create a major health problem in China. Despite being chronic, progressive and potentially fatal, many of these disorders go unidentified and are poorly managed because awareness, infrastructure and genetic help are lacking. In China, the fast development of genomic technologies has not resulted in clinics becoming available to everyone, mainly in underserved rural areas. All healthcare stakeholders, including doctors, labs and genetic counselors, must band together to improve diagnosis, clinician knowledge, newborn screening and genetic counseling. Besides, having a national rare disease registry, investing in translational research and passing laws to support the field can all help patients. The paper argues that China needs to develop a strong, fair and ongoing system for managing rare diseases. It urges policymakers, medical experts, researchers and patient advocate groups to join forces to help people and their families facing rare genetic diseases.
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Evolution of Elderly Healthcare and Pharmaceutical Care: A Historical Overview
Vol.1(1); Pages:27-34. Published on May-2025
Abstract
Learning about geriatric medicine and pharmacy reveals important stages in the history of healthcare, showing how the population, science and society have changed. Because populations across the globe are aging, healthcare providers have identified the distinct and challenging health care requirements of older adults. The overview maps the steps that brought geriatric medicine from ancient times and early curing traditions to the medical specialty it is today. It examines how living longer and having more chronic conditions connected to old age prompted the use of specific medical and drug treatments. There is an increasing focus on personalized care for seniors due to the growth of geriatric pharmacotherapy which concerns adjusting drug dosages to older adults’ bodies and limiting excessive use of many medications. Furthermore, the abstract points out the importance of teamwork between geriatricians and pharmacists to help improve the therapy outcomes and the quality of life of elderly people. By examining these developments in relation to the past and current policies, the book highlights why it is vital to keep innovating, consider ethics and base aging care on research. As a result, this perspective prepares us to guide the future integration of geriatric ideas into total and lasting healthcare systems.
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Pulmonary Arterial Hypertension Patient Profiles: Findings from a New Real-World Data Registry
Vol.1(1); Pages:35-43. Published on May-2025
Abstract
PAH is a severe disease that slowly causes the vascular system to resist pressure, until the right heart gives out and leads to death. Experts must consider the diversity among patients with PAH to achieve better patient outcomes. We studied real-world patient data from an advanced data bank, giving us a full overview of the population, medical care and treatment approaches in individuals with PAH. Repository access to longitudinal records, lab tests, pictures, heart condition reports and medicines enables doctors to see patient medical journeys, diseases they have and how treatments have worked over time. The results demonstrate that the time when patients develop heart failure, their sex balance, what causes it, what its main features are and what their response is to guidelines all differ widely. Crucially, the research points out that certain people and treatment areas are being overlooked and could gain from extra attention. Using this kind of evidence enhances the way PAH is understood and supports tailoring medicine for those with pulmonary hypertension.
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