| (H-L-Arginyl-L-Valyl-L-Isoleucyl-L-Arginyl-L-Alanyl-L-Cysteinyl-L-Leucyl-Glycyl-L-Valyl-Glycyl-L-Leucyl-L-Leucyl-Glycyl-L-Asparaginyl-L-Leucyl-D-Alanyl-Glycyl-L-Lysyl-amino-PEG12-propionic acid)2 (Disulfide bond at Cys) |
Treatment of idiopathic pulmonary fibrosis (IPF) |
| 1,2:5,6-dianhydrogalactitol |
Treatment of Diffuse Intrinsic Pontine Glioma (DIPG) |
| 1-[12-(1 Carboxycyclopropyl) dodecyl] cyclopropane-1-carboxylic acid |
Treatment of Congenital X-linked Nephrogenic Diabetes Insipidus |
| 6-(4-((1s,3s)-1-amino-3-hydroxycyclobutyl) phenyl)-1-ethyl-7- phenyl-1H-pyrido[2,3-b][1,4]oxazin-2(3H)-one L-Tartrate salt |
Treatment of Hereditary Hemorrhagic Telangiectasia |
| a DMD exon 44 skipping PMO and a proprietary cyclic peptide, EEV, that is designed to enable robust muscle tissue uptake, high cellular internalization and endosomal escape of oligonucleotide therapeutics |
Treatment of Duchenne muscular dystrophy |
| a fusion protein comprised of genetically engineered human serine peptidase inhibitor and human immunoglobulin G1 Fc |
Treatment of Netherton Syndrome |
| a genetically modified salmonella comprising of methioninase |
Treatment of hepatocellular carcinoma |
| a modified version of a leucine decarboxylase enzyme from Planctomycetaceae bacterium |
Treatment of Maple Syrup Urine Disease |
| Adenine |
Treatment of Epidermolysis Bullosa |
| Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor VIII Transgene |
Treatment of Hemophilia A |
| anifrolumab |
Treatment of Idiopathic Inflammatory Myopathies |
| azeliragon |
Treatment of Glioblastoma |
| bitopertin |
Treatment of Erythropoietic Protoporphyria |
| cannabidiol |
Treatment of Glioblastoma |
| conditionally-replicative CD40L-expressing adenovirus |
Treatment of malignant glioma |
| fixed-dose combination of potassium citrate and potassium bicarbonate |
Treatment of primary/inherited distal renal tubular acidosis |
| Hemi L-glutarate salt of 7-[(3R,4R)-3-Hydroxy-4-hydroxymethyl-pyrrolidin-1-ylmethyl]-3,5- dihydro-pyrrolo [3,2-d] pyrimidin-4-one |
Treatment of acute lymphoblastic leukemia |
| Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) |
Treatment of Retinitis Pigmentosa |
| Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) |
Treatment of Leber congenital amaurosis |
| Humanized one-armed monoclonal antibody with high affinity for NMDA receptor |
Treatment of anti-N-methyl-D-aspartate (anti-NMDA) receptor encephalitis |
| ifenprodil |
Treatment of Idiopathic Pulmonary Fibrosis |
| Imiquimod |
Treatment of Stage IIB-IV melanoma |
| ketamine hydrochloride |
Prevention of ischemia/reperfusion injury during solid organ transplantation. |
| L-Ergothioneine |
Treatment of Cystinuria |
| Live biotherapeutic product consisting of a single bacterial strain (Leuconostoc citreum [L. citreum]) |
Treatment of Primary Sclerosing Cholangitis |
| luveltamab tazevibulin |
Treatment of pediatric acute myeloid leukemia |
| N-hydroxy-4-((4-((((1R,2S)-2-phenylcyclopropyl) amino)methyl)-1H-pyrazol-1-yl)methyl)benzamide hydrogen bromide |
Treatment of small cell lung cancer |
| N-[(1S)-1-(4- fluorophenyl)ethyl]-4-(1-methyl-1H-pyrazol-4-yl)-N'-(pyrazin-2-yl)pyridine-2,6- diamine maleate |
Treatment of myelofibrosis |
| Recombinant humanized IgG1 anti-Claudin18.2 monoclonal antibody conjugated to MMAE |
Treatment of Pancreatic cancer (PC) |
| Recombinant humanized IgG1 anti-Claudin18.2 monoclonal antibody conjugated to MMAE |
Treatment of Gastric cancer (GC), including cancer of the gastroesophageal junction (GEJ) |
| Self-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 |
Treatment of patients with Spastic Paraplegia Type 50 (SPG50) caused by the AP4M1 gene mutation |
| single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene |
Treatment of hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC) |
| Small molecule inhibitor of PRMT5 (protein arginine methyl transferase 5) |
Treatment of malignant glioma |
| T cell therapeutic product obtained from transduction of chimeric T cell receptor synthetic T cell receptor and antigen receptor (STAR) |
Treatment of Acute Myeloid Leukemia |
| Taldefgrobep Alfa |
Treatment of spinal muscular atrophy |
| Tegavivint |
Treatment of Hepatocellular Carcinoma |
