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.
Chronic pain is a debilitating condition that affects many individuals throughout their daily lives. While it is common to treat chronic pain with pharmaceutical treatments, an approach that has also shown great benefits is the use of integrative medicine, such as massage therapy, osteopathic and spinal manipulation, acupuncture, and yoga. The keywords "integrative medicine," "pain," "chronic pain," and "pain management" with the use of the Boolean operators "AND," "OR," and "NOT" were used to identify relevant studies discussing the effectiveness of alternative medicine in the treatment of chronic pain. Massage therapy uses different forms of pressing, rubbing, and moving of muscles and other soft tissues, and has shown short-term benefits for chronic pain relief. Osteopathic and spinal manipulation is mainly used in treating muscles, tendons, and bone pain due to worn-out joints, torn ligaments, and more. Acupuncture involves penetrating the skin with thin needles which are activated through gentle and specific movements. According to our review, acupuncture and massage therapy are effective for short-term treatment, lasting three to five months for chronic pain. Yoga involves various physical, mental, and spiritual practices or disciplines that have shown beneficial results in the treatment of chronic pain. Combining yoga with physical therapy has shown significant benefits. This review aims to describe the benefits and uses of integrative medicine in the treatment of chronic pain.
Background: Anxiety is a state characterized by somatic, emotional, cognitive, and behavioral components, associated with significant disability. The pharmacotherapy for anxiety remains limited for achievable safety and tolerability of the medicines. Benzodiazepines use associated with side effects like psychomotor impairment and addiction liability. Due to the ADRs associated with antianxiety drugs, the drug trials have focused on screening herbal medicines that are reportedly used in the treatment of anxiety and which have minimal side effects.Methods: The anxiolytic activity was examined by using the elevated plus maze (EPM) and open field test (OFT), forty Albino wistar strain rats of both sex of weighing 120 to 200 g were divided into four groups of ten rats each.. Group 1 received vehicle (normal saline); group 2 received diazepam (1 mg/kg); groups 3 and 4 received BacoMind™, 30 and 60 mg/kg oral, respectively.Results: Rats treated with diazepam (1 mg/kg, p.o.) showed significant (p<0.001) increase in the percentage of open arms entries and time spent whereas, in closed arm the number of entries and time spent were significantly (p<0.05) decreased. Intraperitonial administration of BacoMind™ extract of plant Bacopa monnieri Linn. exhibited significant (p<0.05) increase in the number of open arm entries and time spent with significant (p<0.05) reduction in number of entries and time spent in the closed arm as compared to group 1. BacoMind™ treated rats also produced significant increase in the number of rearings (p<0.05), assisted rearings and number of squares crossed (p<0.01).Conclusions: BacoMind™ extract of plant Bacopa monnieri Linn possess significant anxiolytic activity in the rats. It can be a promising anxiolytic agent.
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