On January 23, 2013, a formal clinical trial to evaluate HPβCD cyclodextrin therapy as a treatment for Niemann–Pick disease, type C was announced by scientists from the NIHs National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). In January 2021, the Sponsor (Mallinckrodt Pharmaceuticals) concluded that the benefit / risk balance for HPβCD cyclodextrin (adrabetadex) for the treatment of neurologic symptoms of NPC was negative, and that the risks associated with the treatment outweigh the potential benefit. Effective immediately, Mallinckrodt recommended that treatment with adrabetadex be discontinued as soon as possible, with the appropriate physician oversight. N-Acetyl-Leucine
N-Acetyl-Leucine is an orally administered, modified amino acid that is being developed as a novel treatment for multiple rare and common neurological disorders by IntraBio Inc (Oxford, United Kingdom).N-Acetyl-Leucine has been granted multiple orphan drug designations from the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of various genetic diseases, including Niemann-Pick Disease Type C. The US FDA has granted IntraBio a Rare Pediatric Disease Designation for N-Acetyl-Leucine for the treatment of NPC.Observational studies in NPC patients have demonstrated the symptomatic, as well as disease-modifying, neuroprotective effect of treatment.
Cefuroxime axetil, sold under the brand name Ceftin among others, is a second generation oral cephalosporin antibiotic. It is an acetoxyethyl ester prodrug of cefuroxime which is effective orally. The activity depends on in vivo hydrolysis and release of cefuroxime tablets. It was patented in 1976 and approved for medical use in 1987. Medical uses
Second generation cephalosporins are more effective in treating Gram-negative bacilli compared to first generation cephalosporins, which have a greater coverage for Gram-positive cocci. Also, it has been reported that cefuroxime is resistant to hydrolysis by β-lactamases produced by Gram-negative bacteria.Some medical uses are:
Upper respiratory tract infections. Lower respiratory tract infections. Urinary tract infections. Skin and soft tissue infections. Gonorrhoea. Early Lyme disease. Bacterial susceptibility
Cefuroxime axetil treats infections against methicillin, oxacillin and penicillin-sensitive bacterial strains. Cefuroxime axetil does not work against enterococci. Gram-positive aerobic microorganisms

Staphylococcus aureus (Methicillin-sensitive only)
Staphylococcus epidermis
Streptococcus pneumoniae (Penicillin-sensitive only)Gram-negative aerobic microorganisms

Haemophilus influenzae
Moraxella catarrhalis
Neisseria gonorrhoeae
Escherichia coli
Proteus mirabilis
Klebsiella pneumoniae (variable activity)

Mechanism of action
Cefuroxime axetil is a second generation cephalosporin that, like penicillins antibiotics, contains a β-lactam ring structure. Cephalosporins works as a bactericidal antibiotic; that by binding to penicillin-binding proteins (PBPs), inhibit the last step of the bacterial cell wall synthesis. Once the β-lactam ring binds to PBPs, cross-linking between peptidoglycan units is inhibited. Pharmacokinetics
Absorption: Once consumed, cefuroxime axetil is converted to the active compound cefuroxime by esterases of mucosal cells in the gastrointestinal tract. Cefuroxime is then released for systematic circulation.