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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 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 inf ... :M09AX13 Casimersen :QM09AX90 Equine stem cells :QM09AX99 Combinations References {{ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Quinine
Quinine is a medication used to treat malaria and babesiosis. This includes the treatment of malaria due to ''Plasmodium falciparum'' that is resistant to chloroquine when artesunate is not available. While sometimes used for nocturnal leg cramps, quinine is not recommended for this purpose due to the risk of serious side effects. It can be taken by mouth or intravenously. Malaria resistance to quinine occurs in certain areas of the world. Quinine is also used as an ingredient in tonic water to impart a bitter taste. Common side effects include headache, ringing in the ears, vision issues, and sweating. More severe side effects include deafness, low blood platelets, and an irregular heartbeat. Use can make one more prone to sunburn. While it is unclear if use during pregnancy causes harm to the baby, treating malaria during pregnancy with quinine when appropriate is still recommended. Quinine is an alkaloid, a naturally occurring chemical compound. How it works as a medicin ... [...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 used either as a nutrient, providing both carbon and nitrogen to the bacteria, or in some patho ... [...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 mut ... [...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 was approved for medical use in Canada in June 2022, and in the United States in August 2023. 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 severe α(1)-antitrypsin deficiency. In 2011, animal studies demonstrated that RARγ agonists, including palovarote ... [...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 the first oral medication approved to treat this disease. 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 by the US Food and Drug Administration (FDA) in August 2020, for the treatment of adults and children two months of age or older. Developed in association with PTC Therapeutics and the SMA Foundation, it is marketed in the US by Genentech, a subsidiary of Roche. Medical uses In the United States, risdiplam is indicated to treat people two months of age and older with spinal muscular atrophy. Adverse ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Onasemnogene Abeparvovec
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time intravenous injection, infusion into a vein. Onasemnogene abeparvovec works by providing a new copy of the gene that makes the SMN1, human SMN protein. The treatment must be accompanied by a course of corticosteroids of at least two months. Common side effects include vomiting and increased liver enzymes. Onasemnogene abeparvovec was first approved for medical use in the United States in 2019 as a treatment for children less than two years old. It was later approved in other jurisdictions with similar scope. The approval scope in certain jurisdictions, including the European Union and Canada, is somewhat different. Medical uses Onasemnogene abeparvovec has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the survival motor neuron 1, ''SMN1'' gene on chromosome 5q and diagnosed pr ... [...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 (DMD). It is an antisense oligonucleotide drug 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 (DMD) 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 DMD patients 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 (nasopharyngitis) and nausea ... [...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. In old ... [...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 pain ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Drisapersen
Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968) is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA. Mechanism of action Duchenne muscular dystrophy (DMD) is caused when a mutation in the dystrophin gene changes the RNA so that it no longer codes for functional dystrophin protein. This usually happens due to a mutation that alters the reading frame of the RNA downstream of the mutation, so-called frameshift mutation. If an exon with an appropriate number of bases lies near the mutation, removing that exon can correct the downstream reading frame, restoring the production of partially functional dystrophin. This is the general strategy used in the design of exon-skipping oligonucleotides for DMD. As t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hydroquinine
Dihydroquinine, also known as hydroquinine, 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. See also * Dihydroquinidine Dihydroquinidine (also called hydroquinidine) is an organic compound, a cinchona alkaloid closely related to quinine. The specific rotation is +226° in ethanol at 2g/100 ml. A derivative of this molecule is used as chiral ligand in the AD-mi ... Dihydroquinidine">Buchler a company focussing solely on cinchona alkaloids as catalysts such as Dihydroquinine or Dihydroquinidine References {{Other drugs for disorders of the musculo-skeletal system Secondary alcohols Phenol ethers Quinoline alkaloids Quinuclidine alkaloids ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ataluren
Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics. Medical use Ataluren is used in the European Union to treat people with Duchenne muscular dystrophy who have a nonsense mutation in the dystrophin gene, can walk, and are more than five years old. Contraindications People who are pregnant or breast feeding should not take ataluren. Adverse effects More than 10% of people taking ataluren in clinical trials experienced vomiting; more than 5% experienced diarrhea, nausea, headache, upper abdominal pain, and flatulence; between 1% and 5% of people experienced decreased appetite and weight loss, high levels of triglycerides, high blood pressure, cough, nosebleeds, abdominal discomfort, constipation, rashes, pain in their arms, legs, and chest muscles, blood in their urine, urinary incontinence, and fever. Interactions Aminoglycosides should not be given to someone taking ataluren, a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
