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NICE

NICE

Onasemnogene abeparvovec is recommended as an option for treating 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of type 1 SMA in babies, only if: they are 6 months or younger, or they are aged 7 to 12 months, and their treatment is agreed by the national multidisciplinary team. It is only recommended for these groups if: permanent ventilation for more than 16 hours per day or a tracheostomy is not needed For babies aged 7 to 12 months, ...

NICE

NICE

Evidence on the efficacy of electrohydraulic lithotripsy for difficult-to-treat bile duct stones is adequate. However, evidence on its safety is limited in quantity. Therefore, it should only be used with special arrangements for clinical governance, consent, and audit or research. Clinicians wishing to do electrohydraulic lithotripsy for difficult-to-treat bile duct stones should: - Inform the clinical governance leads in their healthcare organisation. - Give patients (and their families and ...

NICE

NICE

Acute liver failure is characterised by a rapid (typically in less than 4 weeks) decline in liver function. Causes include poisoning because of alcohol, pharmaceutical or recreational drugs, and viral infection. Less common causes are metabolic disease and acute fatty liver of pregnancy. In this procedure a veno-venous circuit is usually used to perfuse the patient's blood through an extracorporeal whole liver. The aim is to provide metabolic support and prolong survival, to allow time for the ...

NICE

NICE

Lipodystrophy is a rare and heterogeneous group of syndromes characterized by total or partial loss, or absence, of subcutaneous adipose tissue. This results in the accumulation of lipids in abnormal sites such as the liver and muscle. Currently, the condition is managed with lifestyle modifications, cosmetic surgery, and medications to control metabolic disorders, including lipid-lowering medications and antidiabetic therapy. Metreleptin is an analogue of the human hormone leptin, which is ...

NICE

NICE

Urogenital atrophy most often occurs during or after menopause. The lack of the hormone estrogen causes thinning of the tissues around the vaginal area and a reduction in the number of mucus-producing glands. The most common symptoms of the vulva and vagina, including sequelae, pain during intercourse, itching and vaginal discharge. There is an increased vulnerability to inflammation, trauma, and infection. Urogenital atrophy can also cause urinary symptoms, such as urgency to urinate and ...