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Hydrocortisone acetate

Hydrocortisone valerate

Fursultiamine

Compound used for therapy of thiamine deficiency. It has also been suggested for several non-deficiency disorders but has not yet proven useful. Fursultiamine is a vitamin B1 derivative.

Betrixaban

It was selected among all lead compounds due to its low hERG channel affinity while sustaining its factor Xa inhibition capacity [A27286]. … VTE can be manifested as deep vein thrombosis or pulmonary embolism and it is a leading cause of preventable death in hospitalized patients [A27287].

Inotuzumab ozogamicin

Inotuzumab ozogamicin is an antibody-drug conjugate consisting of a humanized IgG4 kappa CD22-targeting monoclonal antibody covalently attached to calicheamicin derivative, N-acetyl-gamma-calicheamicin … [A20352] The antibody portion of the drug binds to CD22 receptors expressed on leukemic B cells and intracellularly releases N-acetyl-gamma-calicheamicin dimethylhydrazide, which produces cytotoxic effects

Dalfampridine

This is the first drug that was specifically approved to help with mobility in these patients. FDA approved on January 22, 2010.

Besilesomab

Besilesomab is a mouse monoclonal antibody labelled with the radioactive isotope technetium-99m for determining the location of inflammation/infection in peripheral bone in adults with suspected osteomyelitis … Utilised only as a diagnostic agent, besilesomab is currently approved by the EMEA for marketing and use in various European countries like Italy, France, Germany, Spain, Portugal, Norway, Sweden, the

Mirdametinib

[L52485] NF1 is an autosomal-dominant genetic condition caused by loss-of-function variants in the _NF1_ tumour suppressor gene,[A265065,A265070] leading to neurofibromin dysfunction and aberrant activation … On February 11, 2025, mirdametinib was approved by the FDA for the treatment of neurofibromatosis type 1 (NF1).

Pitavastatin

[A181090,A181093,A181096,A181427,A181475,A181538] Statins are considered a cost-effective treatment option for CVD due to their evidence of reducing all-cause mortality including fatal and non-fatal CVD … [A181087, A181406] Increasing use of the statin class of drugs is largely due to the fact that cardiovascular disease (CVD), which includes heart attack, atherosclerosis, angina, peripheral artery disease

Nusinersen

An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. … FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.