Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants and children. SMA has been the leading inherited cause of infant death. More specifically, SMA is caused by the absence of the SMN1 gene. In May 2019, the Food and Drug Administration (FDA) approved onasemnogene abeparvovec, SMN1 gene replacement therapy, for all children with SMA younger than two years of age, without end-stage weakness. The objective of the study is to review the safety and efficacy of a novel gene therapy, onasemnogene abeparvovec (Zolgensma), for SMA and assess current challenges for gene therapy. For this, we have conducted a literature search on PubMed, MEDLINE, and Ovid (2019 to 2022) in the English language using the terms SMA, onasemnogene, and gene therapy. The search included articles, websites, and published papers from reputable health organizations, hospitals, and global organizations dedicated to bringing awareness to Spinal Muscular Atrophy. We found the first gene therapy for SMA to be onasemnogene, directly providing the survival motor neuron 1 (SMN1) gene to produce the survival motor neuron (SMN) protein. Onasemnogene is approved by the Food and Drug Administration and has the added benefit of being a one-time dose. On the downside, a major side effect of this treatment is hepatotoxicity. There is substantial evidence that the efficacy of therapy is increased when administered early to children under three months of age. Therefore, we concluded that onasemnogene appears to be an efficacious therapy for younger pediatric patients with SMA type 1. Drug cost and potential hepatotoxicity are major concerns. Long-term benefits and risks have not been determined, but it is more cost-effective and requires less time of treatment compared to the other used drug, nusinersen. Therefore, the combined safety, cost, and effectiveness of onasemnogene abeparvovec make it a reliable treatment option for treating SMA Type 1.

Subclinical hypothyroidism (SCH) or “mild thyroid failure” is defined as elevated serum thyroid-stimulating hormone (TSH) in the presence of normal free thyroxine (T4). The incidence of SCH is estimated at 4.4-8.5% of the general population and occurs more frequently in women. Given that it falls below the diagnostic threshold, SCH is monitored rather than treated. Its management is a common topic of debate as SCH frequently progresses into overt hypothyroidism and is linked to long-term hyperlipidemia, endothelial dysfunction, cardiovascular disease, heart failure, and cerebrovascular disease. Premature hormone administration and lifestyle interventions have been explored as treatment options to mitigate the symptoms of SCH. Our review compares both modalities’ efficacy and potential for standardized clinical practice. A trial of levothyroxine demonstrated significant results in specific SCH demographics, such as patients who are pregnant or trying to conceive, those with goiter, those with thyroid peroxidase (TPO) antibody status, those with steadily increasing TSH, children, and adolescents. All other SCH patients presenting with chronic symptoms may also be reasonably considered for a three- to six-month trial of treatment. Lifestyle modifications through improved sleep hygiene, a diet within the recommended daily allowance (RDA) for iodine and selenium, increased exercise, and smoking cessation also proved efficacious. Our findings indicate that a synergistic approach to treatment is most favorable. Lifestyle modifications neither show adverse effects nor contraindications and can be safely recommended alone or alongside levothyroxine for the treatment of SCH.

The aim of this evidence-based study is to narrate and evaluate the current evidence on recommendations for practicing physicians and other healthcare providers regarding integrative approaches to managing pain in patients with cancer. This review will assess the guideline recommendations and analyze the role of integrative medicine in addressing cancer pain in patients. The literature search highlights relevant studies that will inform evidence-based recommendations for practicing physicians, highlighting their relevance and weaknesses. Acupuncture, massage, and hypnosis have intermediate-strength evidence quality and are moderately recommended for various types of cancer pain. Most of the evidence points to acupuncture being recommended for aromatase inhibitor-related joint pain, hypnosis for procedural pain, and massage for palliative care pain. Other practices with lower-quality evidence include yoga and guided imagery with progressive muscle relaxation, mostly recommended for general cancer pain or musculoskeletal pain. Additionally, music therapy is recommended for procedural or surgical pain. Low-quality or inconclusive evidence was found for other mind-body interventions or natural products. Similarly, there is insufficient evidence to provide recommendations for pediatric patients. Further research is required to enhance our understanding of the role of integrative medicine interventions in caring for cancer patients.

This study investigates the predicting factors of the biochemical response and survival of patients with advanced metastatic prostate cancer who underwent therapy with radioligand lutetium-177 ( 177 Lu)prostate-specific membrane antigen (PSMA), often referred to as [ 177 Lu]Lu-PSMA. This study is a review of the previous literature. This study included articles published in the last 10 years in the English language.According to the literature review, treatment with [ 177 Lu]Lu-PSMA has a positive impact on prostatespecific antigen (PSA) within the first cycle and a negative impact on lymph node metastasis. There is a plausible positive impact on PSA after multiple cycles and performance status and a negative impact on visceral metastasis. In conclusion, the reviews show that treatment with [ 177 Lu]Lu-PSMA in patients with castration-resistant prostate cancer is beneficial in reducing PSA and metastasis.

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