Genetic Disorders
This page contains the most recent discussion threads pertaining to Genetic Disorders, as well as a list of all of the drugs falling within this category.Genetic Disorders Overview
A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polygenic) or by a chromosome abnormality. Although polygenic disorders are the most common, the term is mostly used when discussing disorders with a single genetic cause, either in a gene or chromosome. The mutation responsible can occur spontaneously before embryonic development (a de novo mutation), or it can be inherited f...Read more Genetic Disorders Details
Article is from Wikipedia. All text is available under the terms of the GFDL (GNU Free Documentation License). Source: en.wikipedia.org/wiki/Genetic_disorder
Recent Genetic Disorders Forums:
White pill upside down V 150
its long like capsual its white with a A or upside down V on one side and 150 on the other ## white capsule with an upside down V 150 on one side and blank on the back side. ## I have these pills in my jar of CVS pharmacy chest congestion relief. It's suppose to be 400 mg guaifensin and 20mg dextromehorphan. However, in the save jar I found other pills with TCL 280 that have also been identified as guaifensin... go figure! ## Did you find out what they were? ## Generic mucinex dm. I buy them for my dog
Updated in Kalydeco
its long like capsual its white with a A or upside down V on one side and 150 on the other ## white capsule with an upside down V 150 on one side and blank on the back side. ## I have these pills in my jar of CVS pharmacy chest congestion relief. It's suppose to be 400 mg guaifensin and 20mg dextromehorphan. However, in the save jar I found other pills with TCL 280 that have also been identified as guaifensin... go figure! ## Did you find out what they were? ## Generic mucinex dm. I buy them for my dog
Updated in Kalydeco
Mitochondrial disease
I was Dx with Mitochondrial Myopathy in 9/09 after much testing and labs and finally by muscle biopsy. I was recently told by my neurologist that there had been some research done revealing their effectiveness of Leucovorin in helping to control the duration, intensity and frequency of muscular spasms and cramping, sometimes crippling in its active phases. I began this therapy and within just a few weeks I was having less intensive cramping, the duration was even for less amounts of time. It became more tolerable. It did not do much in controlling the weakness and fatigue I felt but any relief was greatly appreciated, The local pharmacies had to pool their supplies to fill my prescription, I guess my dosage was higher than the usual. It was in a word experimental. I was taking 75 mg eve...
Updated in Leucovorin
I was Dx with Mitochondrial Myopathy in 9/09 after much testing and labs and finally by muscle biopsy. I was recently told by my neurologist that there had been some research done revealing their effectiveness of Leucovorin in helping to control the duration, intensity and frequency of muscular spasms and cramping, sometimes crippling in its active phases. I began this therapy and within just a few weeks I was having less intensive cramping, the duration was even for less amounts of time. It became more tolerable. It did not do much in controlling the weakness and fatigue I felt but any relief was greatly appreciated, The local pharmacies had to pool their supplies to fill my prescription, I guess my dosage was higher than the usual. It was in a word experimental. I was taking 75 mg eve...
Updated in Leucovorin
Tab nitisinone in Indian market
My 48yr Brother is Suffering From Ochronosis. Can We Buy Tab Nitisinone in Indian Market? Please give me the details in this regard. ## Kindly let me know if NITISINONE Tablets are available in India. If yes, then I would also like to know the source for purchase of the same. The Drug is required for a child of 3 years who suffers from heart irregularity. ## please give address & contact details of medicals in india from where Orphadin - Nitisinone drug is available. ## I want nitisinone tablet for my 2 years baby..suffering from tyrosinemia desese ## Is Nitisinone available in India for the treatment of albinism. If tell me the source. ## Is Nitisinone available in India. What is the address of the shop where it is available. Is it use for albinism. What is the price & how it u...
Updated in Nitisinone
My 48yr Brother is Suffering From Ochronosis. Can We Buy Tab Nitisinone in Indian Market? Please give me the details in this regard. ## Kindly let me know if NITISINONE Tablets are available in India. If yes, then I would also like to know the source for purchase of the same. The Drug is required for a child of 3 years who suffers from heart irregularity. ## please give address & contact details of medicals in india from where Orphadin - Nitisinone drug is available. ## I want nitisinone tablet for my 2 years baby..suffering from tyrosinemia desese ## Is Nitisinone available in India for the treatment of albinism. If tell me the source. ## Is Nitisinone available in India. What is the address of the shop where it is available. Is it use for albinism. What is the price & how it u...
Updated in Nitisinone
20 years
Im turning 20 on 2/9 and i have been taking orfadin since i was 6 months old.because i have been diagnosed with tyrosinemia. I was supposed to get a liver transplant but this new mediction saved me from it. I take 48mg twice a day. Doctor prescribed but i feel it is too much. Thoughts? ## I think that in such a situation, with such a dangerous medical issue, that your best and safest bet is to follow your doctor's instructions, because they are going to know more than anyone else. Learn more Orfadin details here. If they've kept you from needing a transplant for this long, I'd say they're doing pretty well, so far. And congratulations to you on such and I hope you have a fantastic 20th birthday. Is there anything else I can help with? ## DO YOU NEED A PRESCRIPTION FOR NI...
Updated in Orfadin
Im turning 20 on 2/9 and i have been taking orfadin since i was 6 months old.because i have been diagnosed with tyrosinemia. I was supposed to get a liver transplant but this new mediction saved me from it. I take 48mg twice a day. Doctor prescribed but i feel it is too much. Thoughts? ## I think that in such a situation, with such a dangerous medical issue, that your best and safest bet is to follow your doctor's instructions, because they are going to know more than anyone else. Learn more Orfadin details here. If they've kept you from needing a transplant for this long, I'd say they're doing pretty well, so far. And congratulations to you on such and I hope you have a fantastic 20th birthday. Is there anything else I can help with? ## DO YOU NEED A PRESCRIPTION FOR NI...
Updated in Orfadin
nitisinone availablity in India
my son is suffering in tyrosinemia type-1st. Treatment is only capsule orfadin (salt, nitisinone) if any body have any information related how i can find this medicen in india or address of any authorized medicare in india, please let me know {edited for privacy}. ## Hi Rouf, Sorry to hear about the condition your son is in. I don't know what the laws are like over in India regarding Orfadin (Nitisinone) being available over-the-counter or via prescription-only (as I am here in the US where it is classified as a prescription drug). But it sounds to me like it may be a good idea to contact the doctor who originally diagnosed him with this ailment and see if he/she knows where to find the medication that was recommended for treating it. You could also just walk into any pharmacy there...
Updated in Nitisinone
my son is suffering in tyrosinemia type-1st. Treatment is only capsule orfadin (salt, nitisinone) if any body have any information related how i can find this medicen in india or address of any authorized medicare in india, please let me know {edited for privacy}. ## Hi Rouf, Sorry to hear about the condition your son is in. I don't know what the laws are like over in India regarding Orfadin (Nitisinone) being available over-the-counter or via prescription-only (as I am here in the US where it is classified as a prescription drug). But it sounds to me like it may be a good idea to contact the doctor who originally diagnosed him with this ailment and see if he/she knows where to find the medication that was recommended for treating it. You could also just walk into any pharmacy there...
Updated in Nitisinone
inf-y actimmune
side effects of this drug and what is it used for ## Actimmune contains a bioengineered formation of interferon gamma. It's a protein that acts as a biological response modifier, it's used to stimulate your immune system. Side effects may include: headache, fever, diarrhea and vomiting. You can learn more about it here: Is there anything else I can help with? ## All of my joints ache and my back never stops hurting is this just part of the side effects or should i be concerned?
Updated in Actimmune
side effects of this drug and what is it used for ## Actimmune contains a bioengineered formation of interferon gamma. It's a protein that acts as a biological response modifier, it's used to stimulate your immune system. Side effects may include: headache, fever, diarrhea and vomiting. You can learn more about it here: Is there anything else I can help with? ## All of my joints ache and my back never stops hurting is this just part of the side effects or should i be concerned?
Updated in Actimmune
Regarding Treatment For Ochronosis
Tab orfaden(Nitisinone)can be used for Alkeptonurea or Patient suffering ochronosis disease approved by FDA America.As per my idea INDIA has got no such drugs to treat this rare genetic disorder decease.May I ask your Medical team to advice me to treat my 48yr Brother Who is having this above disease, for the last 10yr.The said disease is diagnosed by Rheamotologist in INDIA. ## Please meet Dr . Amin .Sanjeev at mumbai
Updated in Nitisinone
Tab orfaden(Nitisinone)can be used for Alkeptonurea or Patient suffering ochronosis disease approved by FDA America.As per my idea INDIA has got no such drugs to treat this rare genetic disorder decease.May I ask your Medical team to advice me to treat my 48yr Brother Who is having this above disease, for the last 10yr.The said disease is diagnosed by Rheamotologist in INDIA. ## Please meet Dr . Amin .Sanjeev at mumbai
Updated in Nitisinone
Tetrabenazine used to treat Parkinsons?
My sister has parkinsons and quite a lot of movement. Has this drug been tried for parkinsons? ## Tetrabenazine is actually contraindicated for the treatment of Parkinson’s [1]. In other words, it might actually worsen the symptoms of Parkinson’s or parkinsonism. Do you have any more questions or information to add? Please post back if you do. References: [1]
Updated in Tetrabenazine
My sister has parkinsons and quite a lot of movement. Has this drug been tried for parkinsons? ## Tetrabenazine is actually contraindicated for the treatment of Parkinson’s [1]. In other words, it might actually worsen the symptoms of Parkinson’s or parkinsonism. Do you have any more questions or information to add? Please post back if you do. References: [1]
Updated in Tetrabenazine
huntington's disease
My 65 yr. old brother has Huntington's disease. Just learned that Tetrabenazine has been approved by the FDA this past year. Is anyone out there being treated with it, your results, price of it, and where can i Purchase it. Thanks... ## This past August, the brand Xenazine (Tetrabenazine) was approved by the FDA. Apparently the process for acquiring a prescription is more complicated than many other medications. You can view the support information for this drug at the manufacturer's web site (Ovation Pharmaceuticals): A quote from their page: "REACH is an integral part of the Ovation Pharmaceuticals commitment to ensuring that patients who can benefit from Xenazine therapy gain access to it as quickly as possible. REACH programs help eliminate administrative and financial b...
Updated in Tetrabenazine
My 65 yr. old brother has Huntington's disease. Just learned that Tetrabenazine has been approved by the FDA this past year. Is anyone out there being treated with it, your results, price of it, and where can i Purchase it. Thanks... ## This past August, the brand Xenazine (Tetrabenazine) was approved by the FDA. Apparently the process for acquiring a prescription is more complicated than many other medications. You can view the support information for this drug at the manufacturer's web site (Ovation Pharmaceuticals): A quote from their page: "REACH is an integral part of the Ovation Pharmaceuticals commitment to ensuring that patients who can benefit from Xenazine therapy gain access to it as quickly as possible. REACH programs help eliminate administrative and financial b...
Updated in Tetrabenazine
Tetrabenazine and FDA approval
Tetrabenazine is a new drug awaiting FDA approval for huntington disease that helps with spasms. What website can I visit for information concerning new medicines awaiting approval from the FDA? ## It says it is already available on a named patient basis, which means your doctor has to contact the company and request it just for you, as it is not yet available on the open market in pharmacies yet. They do have a page with instructions: Tetra Page Tetra Info This page has more info on its use.
Updated in Tetrabenazine
Tetrabenazine is a new drug awaiting FDA approval for huntington disease that helps with spasms. What website can I visit for information concerning new medicines awaiting approval from the FDA? ## It says it is already available on a named patient basis, which means your doctor has to contact the company and request it just for you, as it is not yet available on the open market in pharmacies yet. They do have a page with instructions: Tetra Page Tetra Info This page has more info on its use.
Updated in Tetrabenazine
Genetic Disorders Medications (123 results)
- ACTIMMUNE Interferon Gamma-1b1 discussion
- ADAKVEO Crizanlizumab-tmca
- AFAMELANOTIDE
- AGAMREE Vamorolone
- ALBRIOZA Sodium Phenylbutyrate + Ursodoxicoltaurine
- ALDURAZYME Laronidase
- AMONDYS 45 Casimersen
- AMVUTTRA Vutrisiran
- ARIMOCLOMOL
- AVALGLUCOSIDASE ALFA
- BEROTRALSTAT
- BETAINE
- BIRCH TRITERPENES
- BUROSUMAB
- BYLVAY Odevixibat
- CASIMERSEN
- CERDELGA Eliglustat
- CHOLBAM Cholic Acid
- CHOLIC ACID
- CLOVIQUE Trientine Hydrochloride
- CRIZANLIZUMAB
- CRYSVITA Burosumab
- CUPRID Trientine
- CUVRIOR Trientine Tetrahydrochloride
- CYSTADANE Betaine
- DAYBUE Trofinetide
- DOJOLVI Triheptanoin
- DORNASE ALFA
- DUVYZAT Givinostat Hydrochloride
- ELAPEGADEMASE
- ELEXACAFTOR + IVACAFTOR + TEZACAFTOR
- ELFABRIO Pegunigalsidase Alfa-iwxj
- ELIGLUSTAT
- EMAPALUMAB
- EMPAVELI Pegcetacoplan
- EPLONTERSEN
- ETEPLIRSEN
- EVRYSDI Risdiplam
- EXONDYS 51 Eteplirsen
- FILSUVEZ Birch Triterpenes
- FOSDENOPTERIN
- GALAFOLD Migalastat Hydrochloride
- GAMIFANT Emapalumab-lzsg
- GIVINOSTAT
- GIVLAARI Givosiran Sodium
- GIVOSIRAN SODIUM
- GOLODIRSEN
- INTERFERON GAMMA-1B
- ISTURISA Osilodrostat Phosphate
- IVACAFTOR
- IVACAFTOR + TEZACAFTOR
- JOENJA Leniolisib Phosphate
- KALYDECO Ivacaftor1 discussion
- KOSELUGO Selumetinib Sulfate
- LAMZEDE Velmanase Alfa-tycv
- LARONIDASE
- LENIOLISIB
- LIVMARLI Maralixibat Chloride
- LONAFARNIB
- LUMACAFTOR-IVACAFTOR
- LUMASIRAN
- LUSPATERCEPT
- MARALIXIBAT CHLORIDE
- MEPSEVII Vestronidase alfa-vjbk
- METHYLENE BLUE
- MIGALASTAT HYDROCHLORIDE
- MIPLYFFA Arimoclomol Citrate
- NEDOSIRAN
- NEXVIAZYME Avalglucosidase Alfa-ngpt
- NITISINONE 3 discussions
- NITYR Nitisinone
- NULIBRY Fosdenopterin
- ODEVIXIBAT
- ORFADIN Nitisinone1 discussion
- ORKAMBI Lumacaftor + Ivacaftor
- ORLADEYO Berotralstat Hydrochloride
- OSILODROSTAT
- OXBRYTA Voxelotor
- OXLUMO Lumasiran
- PALYNZIQ Pegvaliase-pqpz
- PEGCETACOPLAN
- PEGUNIGALSIDASE ALFA
- PEGVALIASE
- POZELIMAB
- PROVAYBLUE Methylene Blue
- PULMOZYME Dornase Alfa
- REBLOZYL Luspatercept-aamt
- RELYVRIO Sodium Phenylbutyrate + Taurursodiol
- REVCOVI Elapegademase-lvlr
- RISDIPLAM
- RIVFLOZA Nedosiran Sodium
- SCENESSE Afamelanotide
- SELUMETINIB
- SODIUM PHENYLBUTYRATE + TAURURSODIOL
- SYFOVRE Pegcetacoplan
- SYMDEKO Ivacaftor + Tezacaftor
- SYPRINE Trientine
- TAFAMIDIS
- TETRABENAZINE 3 discussions
- TRIENTINE
- TRIHEPTANOIN
- TRIKAFTA Elexacaftor + Ivacaftor + Tezacaftor
- TROFINETIDE
- URIDINE TRIACETATE
- UROLENE BLUE Methylene Blue
- VAMOROLONE
- VELMANASE ALFA
- VEOPOZ Pozelimab-bbfg
- VESTRONIDASE ALFA-VJBK
- VILTEPSO Viltolarsen
- VILTOLARSEN
- VISTOGARD Uridine Triacetate
- VOSORITIDE
- VOXELOTOR
- VOXZOGO Vosoritide
- VUTRISIRAN
- VYNDAMAX Tafamidis
- VYNDAQEL Tafamidis Meglumine
- VYONDYS 53 Golodirsen
- WAINUA Eplontersen Sodium
- XENAZINE Tetrabenazine
- XURIDEN Uridine Triacetate
- ZOKINVY Lonafarnib