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ATC Code M09
M09A Other drugs for disorders of the musculo-skeletal system M09AA Quinine and derivatives :M09AA01 Hydroquinine :M09AA72 Quinine, combinations with psycholeptics M09AB Enzymes :M09AB01 Chymopapain :M09AB02 Collagenase ''clostridium histolyticum'' :M09AB03 Bromelains :M09AB52 Trypsin, combinations M09AX Other drugs for disorders of the musculo-skeletal system :M09AX01 Hyaluronic acid :M09AX02 Chondrocytes, autologous :M09AX03 Ataluren :M09AX04 Drisapersen :M09AX05 Aceneuramic acid :M09AX06 Eteplirsen :M09AX07 Nusinersen :M09AX08 Golodirsen :M09AX09 Onasemnogene abeparvovec :M09AX10 Risdiplam :M09AX11 Palovarotene :M09AX12 Viltolarsen :M09AX13 Casimersen :M09AX14 Givinostat :M09AX15 Delandistrogene moxeparvovec Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. It is designed to deliver into the body a gene that leads to production of Elevidys micro-dy ... :QM09AX90 Equ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hydroquinine
Dihydroquinine, also known as hydroquinine or DHQ, is an organic compound and as a cinchona alkaloid closely related to quinine. The specific rotation is −148° in ethanol. A derivative of this molecule is used as chiral ligand in the AD-mix for Sharpless dihydroxylation. DHQ also inhibits growth of the parasite ''Toxoplasma gondii'' by inducing mitochondrial membrane damage, but does not disrupt host mitochondrial membrane potential, as well as reactive oxygen species In chemistry and biology, reactive oxygen species (ROS) are highly Reactivity (chemistry), reactive chemicals formed from diatomic oxygen (), water, and hydrogen peroxide. Some prominent ROS are hydroperoxide (H2O2), superoxide (O2−), hydroxyl ... (ROS) generation. See also * Dihydroquinidine References {{Other drugs for disorders of the musculo-skeletal system Secondary alcohols Phenol ethers Quinoline alkaloids Quinuclidine alkaloids Methoxy compounds ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Nusinersen
Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder. Since the condition it treats is so rare, Nusinersen has so-called "orphan drug" designation in the United States and the European Union. Medical uses The drug is used to treat spinal muscular atrophy associated with a mutation in the ''SMN1'' gene. It is administered directly to the central nervous system (CNS) using intrathecal injection. In clinical trials, the drug halted the disease progression. In around 60% of infants affected by type 1 spinal muscular atrophy, it improves motor function. Side effects People treated with nusinersen had an increased risk of upper and lower respiratory infections and congestion, ear infections, constipation, pulmonary aspiration, teething, and scoliosis. There is a risk that growth of infants and children might be stunted. I ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Givinostat
Givinostat, sold under the brand name Duvyzat is a medication used for the treatment of Duchenne muscular dystrophy. It is a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle. The most common side effects include diarrhea, abdominal pain, low platelets (thrombocytopenia), nausea/vomiting, an increase in triglycerides (a type of fat in the body) (hypertriglyceridemia), and fever. Givinostat was approved for medical use in the United States in March 2024. Givinostat is the first nonsteroidal medication approved by the US Food and Drug Administration (FDA) to treat people with all genetic variants of Duchenne muscular dystrophy. The FDA considers it to be a first-in-class medication. Medical uses Givinostat is indicated for the treatment of Duchenne muscular dystrophy in people six yea ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Casimersen
Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death. The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain. Casimersen was approved for medical use in the United States in February 2021, and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to skipping exon 45. Medical uses Casimersen is indicated for the treatment of Duchenne muscular d ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Viltolarsen
Viltolarsen, sold under the brand name Viltepso, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen is a Morpholino antisense oligonucleotide. The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever). Viltolarsen was approved for medical use in the United States in August 2020. After golodirsen was approved in December 2019, viltolarsen is the second approved targeted treatment for people with this type of mutation in the United States. Approximately 8% of people with DMD have a mutation that is amenable to exon 53 skipping. Medical uses Viltolarsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by m ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Palovarotene
Palovarotene, sold under the brand name Sohonos, is a medication used for the treatment of heterotopic ossification and fibrodysplasia ossificans progressiva. It is a highly selective retinoic acid receptor gamma (RARγ) agonist. It is taken by mouth. It was approved for medical use in Canada in June 2022, and in the United States in August 2023. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Palovarotene is indicated for the treatment of heterotopic ossification and fibrodysplasia ossificans progressiva. History Palovarotene is a retinoic acid receptor gamma (RARγ) agonist licensed to Clementia Pharmaceuticals from Roche Pharmaceuticals. At Roche, palovarotene was evaluated in more than 800 individuals including healthy volunteers and patients with chronic obstructive pulmonary disease (COPD). A one-year trial did not demonstrate a significant benefit on lung density in moderate-to-severe emphysema secondary to se ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Risdiplam
Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA). Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier. In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections. Additional adverse events observed in the infantile-onset population included upper respiratory tract infection, pneumonia, constipation and vomiting. Risdiplam was approved for medical use in the United States in August 2020. Developed by Roche in Basel, Switzerland, in association with PTC Therapeutics and the SMA Foundation, it is marketed in the US by Genentech, a subsidiary of Roche. In February 2025, the FDA approved a new tablet formulation of risdiplam. Medical uses In the United States, risdiplam is indicated to trea ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Onasemnogene Abeparvovec
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein. SMA stems from an SMN1 gene mutation, causing SMN protein deficiency vital for motor neuron survival. Onasemnogene abeparvovec, a biologic drug utilizing AAV9 virus capsids containing an SMN1 transgene, is administered to motor neurons, boosting SMN protein levels. Common side effects include vomiting and elevated liver enzymes, while more severe reactions involve liver issues and low platelet count. Developed by AveXis and acquired by Novartis, onasemnogene abeparvovec gained various FDA designations and approvals globally. Controversies included data manipulation concerns and delayed reporting to regulatory agencies. Onasemnogene abeparvovec's price is h ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Golodirsen
Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy. It is an antisense oligonucleotide medication of phosphorodiamidate morpholino oligomer (PMO) chemistry. The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms ( nasopharyngitis) and nausea. Medical uses Golodirsen is indicated for the treatment of Duchenne muscular dystrophy in people who have a confirmed mutation of the ''dystrophin'' gene that is amenable to exon 53 skipping. Mechanism of action Golodirsen has been provisionally approved for approximately 8% of all people with Duchenne muscular dystrophy amenable to exon 53 skipping. It works by inducing exon skipping in the dystrophin gene and thereby increasing the amount of dystrophin protein available to muscle fibers. Adverse effects The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms ( naso ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Eteplirsen
Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Eteplirsen only targets specific mutations and can be used to treat about 14% of DMD cases. Eteplirsen is a form of antisense therapy. Eteplirsen was developed by Steve Wilton, Sue Fletcher and colleagues at the University of Western Australia and commercialized by Sarepta Therapeutics. After a controversial debate surrounding the drug's efficacy, during which two FDA review panel members resigned in protest, eteplirsen received accelerated approval from the US Food and Drug administration in late 2016. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) refused to authorize the use of eteplirsen. Adverse effects The following adverse events were observed in at least 10% of people who received eteplirsen in trials: vomiting, contusion, excoriation, arthralgia, rash, catheter site pai ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chymopapain
Chymopapain (, ''chymopapain A'', ''chymopapain B'', ''chymopapain S'', brand name Chymodiactin) is a proteolytic enzyme isolated from the latex of papaya (''Carica papaya''). It is a cysteine protease which belongs to the papain-like protease (PLCP) group. Because of its proteolytic activity, it is the main molecule in the process of chemonucleolysis, used in some procedures like the treatment of herniated lower lumbar discs in the spine by a nonsurgical method. Structure Primary structure Chymopapain's zymogen is made up of a total of 352 residues, and it has a weight of approximately 23.78kDa. Three different regions can be distinguished inside the precursor's chain. * The first 18 aminoacids act as a sorting signal by indicating the final destination of chymopapain inside the cell when being sorted by the Golgi apparatus. Although this final destination is not fully studied yet, other PLCPs are contained in lysosomes and other acidified vesicles and chymopapain is believe ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Aceneuramic Acid
''N''-Acetylneuraminic acid (Neu5Ac or NANA) is the predominant sialic acid found in human cells, and many mammalian cells. Other forms, such as N-Glycolylneuraminic acid, may also occur in cells. This residue is negatively charged at physiological pH and is found in complex glycans on mucins and glycoproteins found at the cell membrane. Neu5Ac residues are also found in glycolipids, known as gangliosides, a crucial component of neuronal membranes found in the brain. Along with involvement in preventing infections (mucus associated with mucous membranes—mouth, nose, GI, respiratory tract), Neu5Ac acts as a receptor for influenza viruses, allowing attachment to mucous cells via hemagglutinin (an early step in acquiring influenzavirus infection). In the biology of bacterial pathogens Neu5Ac is also important in the biology of a number of pathogenic and symbiotic bacteria as it can be used either as a nutrient, providing both carbon and nitrogen to the bacteria, or in some pathog ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
