Are any results of the cystic fibrosis Phase 2a trials available?
PTC TherapeuticsIn November 2006, we presented data from the PTC124 cystic fibrosis clinical trials in Israel and the U.S. Cystic fibrosis is caused by a lack of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. To determine whether PTC124 was inducing production of active CFTR protein in patients, we measured nasal transepithelial potential difference (TEPD).
Related QuestionsAre any results of the Duchenne muscular dystrophy Phase 2a trials available?
PTC TherapeuticsIn May of 2007, we presented positive preliminary data from a Phase 2a clinical trial of PTC124 in patients with Duchenne muscular dystrophy due to a nonsense mutation. This Phase 2a multi-site, open-label, dose-ranging clinical trial has evaluated muscle dystrophin expression in patients with nonsense-mutation-mediated Duchenne muscular dystrophy. Blood levels of muscle-derived creatine kinase have been measured as assessments of muscle integrity.
Related QuestionsWhat is cystic fibrosis?
Cystic Fibrosis Foundation - Frequently Asked QuestionsCystic fibrosis is a life-threatening disease that causes mucus to build up and clog some of the organs in the body, particularly in the lungs and pancreas. When mucus clogs the lungs, it can make breathing very difficult. The thick mucus also causes bacteria (or germs) to get stuck in the airways, which causes inflammation (or swelling) and infections that leads to lung damage. Mucus also can block the digestive tract and pancreas.
Related QuestionsHow do people get cystic fibrosis?
Cystic Fibrosis Foundation - Frequently Asked QuestionsCystic fibrosis is a genetic disease. That means people inherit it from their parents through genes (or DNA), which also determine a lot of other characteristics including height, hair color and eye color. Genes, found in the nucleus of all the body's cells, control cell function by serving as the blueprint for the production of proteins. The defective gene that is responsible for causing cystic fibrosis is on chromosome 7.
Related QuestionsWho gets cystic fibrosis?
Cystic Fibrosis Foundation - Frequently Asked QuestionsApproximately 30,000 people in the United States have cystic fibrosis. An additional ten million more—or about one in every 31 Americans—are carriers of the defective CF gene, but do not have the disease. The disease is most common in Caucasians, but it can affect all races. The severity of cystic fibrosis symptoms is different from person to person.
Related QuestionsIs cystic fibrosis fatal?
Cystic Fibrosis Foundation - Frequently Asked QuestionsCurrently, there is no cure for cystic fibrosis. However, specialized medical care, aggressive drug treatments, and therapies, along with proper CF nutrition, can lengthen and improve the quality of life for those with CF. The best way for people with cystic fibrosis to fight their disease is to work with their medical caregivers at a CF Foundation-accredited care center. The care center partners with people who have CF to help keep them in the best health possible.
Related QuestionsSince the Phase 2a data are positive, will Phase 2b/Phase 3 trials still be necessary?
PTC TherapeuticsThe Phase 2a trials are not designed to assess the efficacy of PTC124 or to compare the efficacy to other therapies. Because they are short in duration, they also cannot examine the safety of longer-term administration of PTC124. The Phase 2a studies form the basis for the development of longer-term Phase 2b/Phase 3 trials.
Related QuestionsPhase III Clinical Trials ' Is It Better Than What's Already Available?
FAQ's - NCCC National Cervical Cancer CoalitionTreatments that show a certain level of effectiveness in phase II studies usually must go through one more stage of testing before being approved for general use. Phase III clinical trials compare the safety and effectiveness of the new treatment against the current standard treatment. Phase III clinical trials require a large number of patients, usually at least several hundred. These studies are generally conducted in many places across the country (or even across the world) at the same time.
Related QuestionsWhat are the goals of the PTC124 Phase 2 studies in cystic fibrosis and Duchenne muscular dystrophy?
PTC TherapeuticsPhase 2a studies are typically conducted on a relatively small number of patients, with the goal of determining drug activity, evaluating short-term side effects, and assessing pharmacokinetics in patients. The duration of several of the initial Phase 2a studies has been based on knowledge that PTC124 is safe when given for up to 28 days to animals and because it has been expected that early drug effects in patients might be observed within this duration of treatment.
Related QuestionsCystic Fibrosis - Questions & AnswersCystic Fibrosis (CF) is an inherited disease that causes the glands that make mucous, saliva and sweat to not work properly. The mucous is sticky instead of thin and slippery and chokes the lungs and can block openings in other major organs, which makes it difficult to break down and absorb food. CF can cause permanent liver damage.Related Questions
Quality Health | Cystic FibrosisCystic fibrosis is a disease that causes mucus in the body to become thick, dry, and sticky. This glue-like mucus builds up and causes problems in many of the body's organs, especially the lungs and the pancreas . People who have cystic fibrosis can have serious breathing problems and lung disease. They can also have problems with nutrition, digestion, growth, and development. There is no cure for cystic fibrosis and the disease generally gets worse over time.Related Questions
What is Cystic Fibrosis and Male Infertility?
Untitled DocumentMen who have cystic fibrosis often have a congenital anomaly in the male genital tract. The vas deferens, the tube connecting the testicle and epididymis to the ejaculatory duct, is congenitally absent. This makes it impossible for the sperms to pass through the penis. Using testicular sperm aspiration, the urologist can obtain sufficient sperm to allow excellent success with IVF and ICSI (intracytoplasmic sperm injection).
Related QuestionsWhat were the results of the ISA247 Phase 2a kidney transplantation trial?
Isotechnika - For Investors - Frequently Asked QuestionsThe primary endpoint of the trial was to demonstrate that stable kidney transplant patients receiving mix-ISA247 experienced no negative change in kidney function when compared to patients on cyclosporine (Neoral®). The secondary endpoint of the study was to measure the pharmacodynamics and pharmacokinetics of mix-ISA247 in renal transplant patients. All of these endpoints were achieved.
Related QuestionsWhy not add screening for cystic fibrosis?
Texas Department of State Health Services - FAQs about Newbo...The 79th Texas Legislature in 2005 mandated that DSHS expand to the ACMG-recommended panel of disorders as funding allowed and appropriated $3.1 million. The startup funding allows 20 additional disorders to be added to the screening panel. Cystic fibrosis startup would require an additional $2 million to $3 million.
Related QuestionsHow Common is Cystic Fibrosis?
Cystic Fibrosis - Questions & AnswersCF is one of the most common inherited diseases of Caucasians (whites). CF occurs in one of every 3,200 live Caucasian births. It occurs in one in every 15,000 live African-American births. CF is uncommon in Asians and most Native American tribes. It is being seen more often in Hispanics. CF affects almost every race. About 1,000 new cases of CF are diagnosed each year.
Related QuestionsCan I "Catch" Cystic Fibrosis?
Cystic Fibrosis - Questions & AnswersNo. CF is a disease that a person is born with. It comes from a defective gene from each parent. Genes are the main unit of heredity. Not all people carry the defective gene that causes CF, so if one parent has the gene and the other does not, the child will not have CF. You cannot control which genes get passed to your child.
Related QuestionsWhat Are the Symptoms of Cystic Fibrosis?
Cystic Fibrosis - Questions & AnswersPeople with CF can have several symptoms including very salty-tasting skin; steady coughing at times with phlegm; wheezing or shortness of breath; a huge appetite but remains thin; and large, greasy stools. These symptoms are different from person to person.
Related QuestionsHow Is Cystic Fibrosis Diagnosed?
Cystic Fibrosis - Questions & AnswersThe main test for CF is called the sweat test and it measures the amount of salt in sweat. Starting in 2002, all babies born in New York State are tested at birth as part of a series of tests on newborn babies. If there is reason to think a child has CF, the test should be done in an accredited CF Care Center.
Related QuestionsHow Is Cystic Fibrosis Treated?
Cystic Fibrosis - Questions & AnswersThere are many treatments for the symptoms and CF related problems. Treatment is aimed at preventing lung infections, reducing the amount and thickness of mucous in the lungs, improving airflow and maintaining nutrition.
Related QuestionsIs There a Cure For Cystic Fibrosis?
Cystic Fibrosis - Questions & AnswersThere is no cure for CF at this time, but ongoing research has led to advances in treatment, which have significantly improved the lives of many CF patients. New discoveries in the treatment of CF have helped people with the disease live longer. Twenty years ago, only half the people with CF lived beyond age 21, but today half the people with CF live past 35 years of age.
Related QuestionsHow Do We Inherit Cystic Fibrosis?
Open Directory - Science: Biology: Genetics: Eukaryotic: Animal: Mammal: Human: Testing and Counseling EBI, the European Bioinformatics Institute (EMBL Outstation, Hinxton, UK) serving the bioinformatics community
Related QuestionsWhere can I find more information about Cystic Fibrosis and its causes?
Pseudomonas Genome Database - Frequently Asked QuestionsVisit the Cystic Fibrosis Foundation website for information and links to further resources on this disease. For more detailed information, including discoverer Riordan et al., please read the following articles at NCBI. PathoGenesis Corporation, who played a major role in sequencing the first P. aeruginosa genome, is now a part of Chiron and continues its interest in the development of new anti-Pseudomonad drugs.
Related QuestionsWhat causes cystic fibrosis?
Quality Health | Cystic FibrosisCystic fibrosis is a genetic disorder. A child must inherit a specific gene from both parents to get cystic fibrosis.
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