Gene therapy for spinal muscular atrophy: the Qatari experience.

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by hypotonia, progressive muscle weakness, and wasting. Onasemnogene abeparvovec (Zolgensma®) is a novel gene therapy medicine, FDA-approved in May 2019 for the treatment of SMA. This study aimed to describe Qatari experience with onasemnogene abeparvovec by reviewing the clinical outcomes of 9 SMA children (7 SMA type 1 and 2 with SMA type 2) aged 4‒23 months treated between November 2019 and July 2020. Children <2 years with 5q SMA with a bi-allelic mutation in the SMN1 gene were eligible for gene therapy. Liver function (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and total bilirubin), platelet count, coagulation profile, troponin-I levels, and motor scores (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND]), were regularly monitored following gene therapy. All patients experienced elevated AST or ALT, two experienced high prothrombin time, and one experienced elevated bilirubin; all of these patients were asymptomatic. Furthermore, one event of vomiting after infusion was reported in one patient. Significant improvements in CHOP INTEND scores were observed following therapy. This study describes the short-term outcomes and safety of onasemnogene abeparvovec, which is well tolerated and shows promise for early efficacy.

Fast microwave-assisted sorption of heavy metals on the surface of nanosilica-functionalized-glycine and reduced glutathione.

Two eco-friendly nanosorbents have been designed and synthesized via surface crosslinking of nanosilica (N-Si) with glycine (Gly) and reduced glutathione (GSH) to produce (N-Si-Gly) and (N-Si-Glu) using crosslinking reagent and sonochemical reactions, respectively. An investigation was performed to search selectivity of nanosorbents via microwave-assisted removal of Ni(II)/Cu(II)/Cd(II)/Pb(II) to affirm green and fast technique. The microwave-assisted removal values of Ni(II), Cu(II), Cd(II) and Pb(II) were observed at 850, 2100, 3500 and 2150 µmol g-1, respectively utilizing 10 mg of (N-Si-Glu) and 25.0 s heating, while those corresponded to 750, 1800, 2500 and 1850 µmol g-1, respectively by using (N-Si-Gly). The microwave-assisted removal processes were more fitted to Freundlich compared to Langmuir isotherm except in case of Pb(II). The high percent removal of Cd(II) and Pb(II) ions exceed 95% from the second run in real wastewater samples indicating the efficiency of N-Si-Glu in the uptake of these metals utilizing microwave-assisted sorption technique.

Removal of copper (II) from aqueous solutions by flotation using polyaluminum chloride silicate (PAX-XL60 S) as coagulant and carbonate ion as activator.

Flotation is a separation technology for removing toxic heavy metal ions from aqueous solutions. Here a simple and rapid flotation procedure is presented for the removal of copper(II) from aqueous solutions. It is based on the use of polyaluminum chloride silicate (PAX-XL60 S) as coagulant and flocculent, carbonate ion as activator and oleic acid (HOL) as surfactant. Both ion and precipitate flotation are included depending on the solution pH. Ion and precipitate flotation in the aqueous HOL-PAX-XL60 S-Cu2+-CO3(2-) system gave powerful preferential removal of Cu2+ (F -100%) over the HOL-PAX-XL60 S-Cu2+ system containing no CO3(2+) ion (F approximately 86%). The role of CO3(2-) ion is also evident from decreasing the dose of PAX-XL60 S from 700 mg l(-1) to 200 mg l(-1). The other parameters, influencing the flotation process, namely: metal ion, surfactant and PAX-XL60 S concentrations, ionic strength, temperature and foreign ions were examined. Moreover, the procedure was successfully applied to recover Cu2+ ions from different volumes up to 11 and from natural water samples.

Studies on Paederus alfierii Koch (Coleoptera:Staphylinidae) with special reference to the medical importance.

In Egypt, the rove beetle; Paederus alfierii Koch, is an active predator of several insects pests attacking a wide variety of cultivated plants as maize, cotton, clover ... etc. On the other hand, members of genus Paederus contain pederin (potent toxin) which in contact with human skin causes a necrotizing lesion (dermatitis linearis) and with the eye causes conjunctivitis. After the sudden flood of the year 1994, this beetle attacked a factory and about forty factors suffered a form of contact dermatitis and conjunctivitis. The majority of such factors needed hospitalization. To fill the gap in the knowledge of medical importance on such a predator, the present investigation was aimed. The aqueous extract from twenty wild collected adults (0.0101 gm) was tested on different groups of albino mice. No cutaneous reaction was seen macroscopically. On the other hand, another group of mice (3/4) showed mild conjunctival congestion faded on the next day. However, these experimental findings do not indicate the innocence of this rove-beetle from being harmful to man. The discussion was given on the light of work done abroad.

A comparison of cefaclor and ampicillin in the treatment of respiratory infection in elderly in-patients.

Fifty patients admitted to a geriatric ward with respiratory tract infection were randomly allocated to treatment with 250 mg cefaclor 8-hourly or 250 mg ampicillin 6-hourly. Assessment of response was performed by nursing and medical staff independently and it was found that the nurses tended to record changes in overall condition earlier than did the doctors. The failure rate was 15%, with no difference between the two treatments. The significance of this in relation to the different pharmacokinetic properties of these antibiotics is discussed.