Latest news with #Miplyffa
Medical News Today
04-08-2025
- Health
- Medical News Today
Miplyffa (arimoclomol)
Miplyffa is a brand-name oral capsule. It's prescribed for symptoms that affect the brain, spinal cord, and nerves in people with a rare genetic condition. Miplyffa contains the active ingredient arimoclomol. Specifically, the Food and Drug Administration (FDA) has approved Miplyffa to treat symptoms that affect the brain, spinal cord, and nerves in people with Niemann-Pick disease type C. It's prescribed for adults and children ages 2 years and detailsYou'll find key information about Miplyffa here:Drug class: heat shock protein stimulatorDrug form: oral capsuleGeneric available? noPrescription required? yesControlled substance? noMiplyffa genericMiplyffa is available only as a brand-name medication. It's not currently available in generic form.A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics usually cost less than brand-name dosageThe Miplyffa dosage your doctor prescribes will depend on several factors. These include:your body weight in kilograms (kg)other medical conditions you may haveFor reference, 1 kg is about 2.2 pounds (lb).The following information describes dosages that are commonly used or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your form and strengthsMiplyffa comes as an oral capsule. It's available in four strengths:47 milligrams (mg)62 mg93 mg124 mgDosage for Niemann-Pick disease type CMiplyffa is prescribed to treat symptoms that affect the brain, spinal cord, and nerves caused by Niemann-Pick disease type C. It's prescribed for adults and children ages 2 years and typical dosage of Miplyffa depends on body weight as outlined below:8 kg to 15 kg (about 17 lb to 33 lb): 47 mg three times dailymore than 15 kg to 30 kg (about 33 lb to 66 lb): 62 mg three times dailymore than 30 kg to 55 kg (about 66 lb to 121 lb): 93 mg three times dailymore than 55 kg (about 121 lb): 124 mg three times dailyYour doctor may prescribe a different dosage depending on certain factors, such as your kidney function. If you have questions about the Miplyffa dosage that's right for you, talk with your taking MiplyffaYou'll find information about key dosage issues here:When to take: You should take Miplyffa three times daily, around the same time each day. This helps keep a steady level of the drug in your body so the drug can work you miss a dose: If you miss a dose of Miplyffa, skip it. Then take your next dose at the regular Miplyffa with food: You can take Miplyffa with or without splitting, or chewing Miplyffa: If possible, Miplyffa capsules should be swallowed whole. But if you have trouble swallowing capsules, you can open them. Sprinkle the contents onto 15 milliliters (mL) of apple juice, water, or a soft food such as yogurt or applesauce. Take the mixture right away. If you use a feeding tube, you can mix the capsule contents with 20 mL of water and give the mixture through the tube. Be sure to flush the tube with 5 mL of water afterward. Length of use: Miplyffa is meant to be a long-term treatment. If you and your doctor determine that Miplyffa is safe and effective for you, you'll likely take it long of time to work: Miplyffa starts to work after your first dose. Your doctor will monitor your condition to determine how well the drug is side effectsMiplyffa can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking Miplyffa. These lists do not include all possible side more information about the possible side effects of Miplyffa, talk with your doctor or pharmacist. They can give you tips on how to manage any side effects that may be concerning or After the Food and Drug Administration (FDA) approves a drug, it tracks and reviews side effects of the medication. Sharing your experience helps make medications safer for everyone by giving doctors and researchers more information about how the drug works in real life. If you develop a side effect while taking Miplyffa and want to tell the FDA about it, visit MedWatch or call side effectsHere is a partial list of mild side effects of Miplyffa. To learn about other mild side effects, talk with your doctor or pharmacist, or view Miplyffa's prescribing side effects of Miplyffa can include:diarrheaheadachehivesloss of appetiteseizuretremorupper or lower respiratory tract infectionweight lossMost of these side effects may go away within a few days to a couple of weeks. However, if they become more severe or don't go away, talk with your doctor or side effectsSerious side effects from Miplyffa aren't common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency phone number if your symptoms feel life threatening or you think you're having a medical side effects can include high creatinine levels, which may affect how kidney function is REACTIONFor some people, Miplyffa can cause an allergic general, symptoms of an allergic reaction can be mild or to manageFor mild allergic reaction symptoms, such as a mild rash, call your doctor right away. They may recommend treatments to help manage your symptoms. They'll also let you know whether you should keep taking the severe allergic reaction symptoms, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms require immediate medical care because they can become life threatening. If you've had a serious allergic reaction to Miplyffa, your doctor may recommend a different medication usesThe following information describes Miplyffa's approved use by the Food and Drug Administration (FDA).Miplyffa for Niemann-Pick disease type CMiplyffa is approved to treat symptoms that affect the brain, spinal cord, and nerves in people with Niemann-Pick disease type C (NPC). It's prescribed for adults and children ages 2 years and is a rare genetic condition that affects how the body stores and removes cholesterol and other fats. Over time, these fats build up in the brain and other parts of the body. This can lead to worsening symptoms that may affect the brain, spinal cord, and nerves. Symptoms of NPC may include seizures and difficulty walking, balancing, speaking, or this purpose, Miplyffa is typically prescribed with miglustat (Opfolda, Yargesca, others). Together, these medications may help slow the worsening of NPC learn more about Miplyffa for NPC, talk with your and childrenMiplyffa is approved to treat NPC in children ages 2 years and learn more about Miplyffa for children with NPC, talk with your child's interactionsMiplyffa can interact with several other medications. However, the drug isn't known to interact with any supplements or taking Miplyffa, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential with medicationsHere is a list of medications that can interact with Miplyffa. This list does not contain all drugs that may interact with Miplyffa. If you have questions about drug interactions that may affect you, ask your doctor or that can interact with Miplyffa• cisplatin (Kemoplat)• cimetidine (Tagamet HB)• ipratropium (Atrovent HFA)• lamivudine (Epivir)• metformin (Glumetza)Alcohol interactionAlcohol is not known to interact with Miplyffa. If you have questions about whether it's safe to drink alcohol while taking Miplyffa, talk with your doctor or cost and savingsAs with all medications, the cost of Miplyffa can vary. The actual price you'll pay depends on your insurance plan, your location, and the pharmacy you and insurance assistance: If you need financial support to pay for Miplyffa or help understanding your insurance coverage, help is available.A program called AmplifyAssist is available for Miplyffa. For more information and to find out if you're eligible for support, call 888-668-4198 or visit the program version: Miplyffa is not available in a generic form. A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics tend to cost less than brand-name drugs.»Learn more about saving money on drugsOther drugs are available that can treat Niemann-Pick disease type C. Examples include levacetylleucine (Aqneursa) and miglustat (Opfolda, Yargesca, others).Some drugs may be a better fit for you than others. If you're interested in finding an alternative to Miplyffa, talk with your doctor. They can tell you about other medications that may work well for to consider when taking Miplyffa If you can become pregnant, consider the following information about pregnancy, fertility, birth control, and and pregnancy It may not be safe to take Miplyffa during pregnancy. This is based on the results of animal studies, which don't always predict what will happen in humans. If you're planning a pregnancy or can become pregnant, talk with your doctor before taking this medication. Your doctor may suggest birth control options to use during treatment with and fertilityIn animal studies, Miplyffa decreased female and male fertility (the ability to conceive). However, animal studies don't always predict what will happen in starting Miplyffa treatment, talk with your doctor about the drug's possible effects on fertility. They can recommend whether Miplyffa is the right treatment and birth controlMiplyffa may not be safe to take during pregnancy. If you're sexually active and you or your partner can become pregnant, talk with your doctor about your birth control options while you're taking The use of binary terms such as 'male' and 'female' or 'men' and 'women' in this article reflects the language of the sources we've used. Unless otherwise noted, it's unclear whether the research we reference included participants with expansive gender females using MiplyffaDoctors may advise females who can become pregnant to use birth control during Miplyffa treatment. If you're a female taking Miplyffa, talk with your doctor about your birth control males using MiplyffaIf you're a male with a sexual partner who can become pregnant, talk with your doctor about your birth control needs during Miplyffa treatment. Miplyffa and breastfeedingIt's not known whether Miplyffa should be taken while breastfeeding. If you're currently breastfeeding or planning to do so, talk with your doctor before taking this Miplyffa worksMiplyffa's mechanism of action (how it works) is not fully understood. Miplyffa is used to treat Niemann-Pick disease type C, which is a rare genetic condition that can cause fats to build up in the body. This buildup may lead to symptoms that affect the brain, spinal cord, and nerves. Miplyffa is thought to help slow the worsening of these you have other questions bout how Miplyffa works, talk with your doctor or precautionsCertain factors or conditions could prevent your doctor from prescribing Miplyffa due to the risk of harm. These are known as contraindications. However, Miplyffa does not have any known taking Miplyffa, discuss your health history with your doctor. Miplyffa may not be suitable for you if you have specific medical conditions or other health factors. Be sure to talk with your doctor if any of the following apply to you:kidney problemsprevious allergic reaction to this or a similar drugpregnancybreastfeedingNote: For more information about the potential negative effects of Miplyffa, see the 'Miplyffa side effects' Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.
CBS News
25-03-2025
- Health
- CBS News
Minnesota mom goes on quest for answers when teen son develops mysterious illness
When a 17-year-old Minnesota boy got sick with a mysterious illness, his mom went on a quest for answers. Connor Martin, of Champlin, stays busy. "I play violin. Right now, I play softball," said Connor Martin. He also loves going to Minnesota Twins games to see his favorite players. But a little more than three years ago, Martin and his family were thrown a curveball. "It just felt like he just was sort of slowing down and I just noticed his reaction time was slower than usual, he was perhaps more clumsy than usual," said mother Jessica Martin. His bloodwork and other tests were normal, but Jessica Martin kept pushing for answers to her son's medical mystery. He was quickly referred to children's neurologist Dr. Jessica Goldstein, at M Health Fairview Masonic Children's Hospital. "There's a lot of detective work. Really looking for clues or key elements that kind of point you in one direction or the other," said Goldstein. Within a few months, there was a diagnosis: Niemann-Pick disease type C (NPC). "It was terrifying," said Jessica Martin. NPC is a rare, inherited, progressive and fatal neurodegenerative disease. Jessica Martin pushed for her son to be part of a clinical trial for a new drug called Miplyffa. Since starting the medicine in late 2022, Connor Martin has made improvements. "It really did stabilize things for him pretty quickly," said Goldstein. "We've seen improvement in swallowing, in speech, in energy level." "The medicines have definitely given us more time and more memories and more opportunities to just enjoy our child," said Jessica Martin. Which, for Connor Martin, includes being in the stands for Twins opening day. "We have an OK team this year," said Connor Martin. And his mom will continue to swing for a cure. "For these rare diseases, gene therapy is really the endgame," said Jessica Martin. "These treatments are hopefully going to buy us all the time we need to get there."
