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Dendritic cells vaccine

With a better safety profile than inactivated and live-attenuated virus and virus-vector vaccines, the recombinant chimeric DC vaccine reduces the risk of virulence recovery, pre-existing anti-vector immunity … The recombinant chimeric DC vaccine is a subunit-based vaccine candidate investigated for safety and effectiveness against SARS-CoV-2-induced pneumonia.

Sodium fluorophosphate

Sodium monofluorophosphate is used in OTC dental preparations to help protect against cavities.

Viltolarsen

Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive ambulatory, pulmonary, and cardiac function … links between nucleotides are replaced with a phosphorodiamidate linkage.

Pegvaliase

The primary goal of lifelong treatment of PKU, as recommended by the American College of Medical Genetics and Genomics (ACMG) guidelines, is to maintain blood Phe concentration in the range of 120 µmol … /L to 3690 µmol/L [A33286].

Actelion-1

Actelion-1 is a tissue-targeting Endothelin Receptor Antagonist that has been discovered by Actelion Pharmaceuticals.

Sodium phosphate, dibasic, unspecified form

Rituximab

FDA in 1997 as a single agent to treat patients with B-cell Non-Hodgkin's Lymphoma (NHL) [L4811], however, has now been approved for a variety of conditions [FDA label]. … Rituximab is a genetically engineered chimeric murine/human monoclonal antibody directed against the CD20 antigen found on the surface of normal and malignant B lymphocytes.

Onasemnogene abeparvovec

muscular atrophy (SMA) and a specific mutation in the survival motor neuron 1 (SMN1) gene. … Developed by AveXis, a Novartis company,[A187253] onasemnogene abeparvovec is commonly marketed as Zolgensma®, which is available as a single-dose intravenous infusion.

Protamine sulfate

Despite the relative importance of protamine sulfate's medical indication, the medication can notoriously cause a variety of potentially rare but genuinely severe adverse effects that include systemic … recovery of a patient after the procedure [A174778, A174781, L5371].