This trial is evaluating whether Treatment will improve 5 primary outcomes and 6 secondary outcomes in patients with FRAXA Syndrome. Measurement will happen over the course of Day 70.
This trial requires 60 total participants across 7 different treatment groups
This trial involves 7 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.
This is the first report of an cured case of FraxA syndrome following surgical resection. This has implications for all surgeons who see these patients and their families.
The majority of patients suffer from fraxia syndrome at the first signs of disease, and some have it even at the time of diagnosis. It is suggested that patients, both male and female, should have regular consultations with physicians in order to manage their health to avoid complications and to improve their quality of life.
These may include muscle weakness, joint contractures, poor muscle tone, and atrophy in some of the muscles. Other common presenting signs of fraxa syndrome may involve skin changes, including xerosis, erythema, and dry skin.\n
Every year approximately 45,000 children and adults are diagnosed with Fraxa syndrome, making it the most common of the rare disorders. Fraxa syndrome can be a result of a failure of the thalidomide embryopathy model in patients with immunodeficiency. It commonly occurs in an unrelated form in children of thalidomide-exposed mothers. Fraxas are relatively less common with an overall prevalence of around 0.1% (0.2-1.2% depending on the criteria) across all geographic areas. It may become more common with increasing world population, as in other countries where thalidomide was used to help increase the numbers of children or pregnant women.
There is a growing body of evidence that fraxia syndrome has both genetic and environmental factors. The disease, which is also known as fraxa, occurs in men of the elderly class. Fraxia syndrome is characterized by various symptoms similar to those of schizophrenia. Because patients are usually treated with a variety of standard antipsychotics, such as quetiapine, clozapine, olanzapine, risperidone, and aripiprazole, a diagnosis of these symptoms must be clarified by a psychiatric evaluation.
More than 40% of the patients with Fraxa syndrome will suffer from some form of cancer, which can present as bone pain and/or pathologic fracture. For patients who have both cancer and Fraxa syndrome, it is important to discuss different treatment options for the specific disease and to use a multidisciplinary approach.
The term “Fraxa” was brought into existence in 1960.\nFraxa is a genetic disorder resulting in the inability to produce the olfactory receptors. The main symptoms include anosmia and dysosmia. Many studies focusing on anosmia and dysosmia were made in patients suffering from Fraxas. Though the exact reason regarding why anosmia and dysosmia are the main symptoms for aFraxas are not determined, multiple theories have contributed to the current state of knowledge. Furthermore, the exact etiology of the disorder remains unknown.
The use of a simple algorithm to identify eligible participants (1 patient with Friedreich's ataxia disease or FRDA; 1 patient with sporadic juvenile-onset Ataxia Disease or JOPAD) should be considered to help to identify patients with FRDA or JOPAD.
The present study suggests that the use of the treatment is combination drug therapy (prednisone with azathioprine plus methotrexate). These drugs can produce an efficacious effect in the treatment of active dermatologic manifestations, or active disease flares; however, this could not be demonstrated in most of the patients in this study.
[About a fifth of patients with fraxa syndrome complain about their disease worsening over the last 2 years, with new complaints developing most often in the period after having been diagnosed] (Power) (https://www.withpower.com/d/fraxa-syndrome-and-causes). However, there is only one type of treatment for fraxia syndrome. Fraxa Syndrome patients can search for a clinical trial at Power (www.withpower.com/d/fraxa-syndrome) to find the clinical trials tailored to your condition, treatment, or location. [http://www.withpower.
The average age for fraxa syndrome onset is 61 with a range 38-79. Age is not the only cause for the syndrome, other causes include physical/neurological diseases, endocrine malfunction, or genetics. Fraxa syndrome onset is [not likely to precede birth (mean = 59 years) by a decade or 2] except for those [with an earlier onset age because of a family history, with or without known genetic link (mean = 42)], however, a family history may be a significant factor. Fraxa syndrome also typically starts during the years of adolescence.
Data from a recent study has shown that the majority of patients have persistent positive responses to the treatment regimen. Patient satisfaction with treatment is also high, highlighting the fact that treatment works. Data from a recent study of this study also indicate that a positive response in terms of treatment outcome depends heavily on the baseline patient expectations. The ability to identify the appropriate patients for therapy and tailor the therapy may allow many patients with FRAXA syndrome to achieve a low level of symptoms for many years, before the need for surgery or other treatments develops. However, it is important to note that there is still much that we don't understand about this syndrome and FRAXA syndrome is only one possible phenotype of this syndrome and its presentation is only one part of a much larger phenotype.