软骨发育不全
软骨发育不全 is a rare genetic bone growth condition characterized by disproportionate short stature, curvature of the spine and an enlarged head (macrocephaly).
Our thoughtful approach to clinical trial design enables us to move quickly and efficiently while prioritizing the safety of participants.
Clinical trials evaluate the safety and 功效 of investigational medicines and are required to support regulatory approval of new medications. 在药物被批准后, clinical trials also play an important role in helping further understand the effects of treatment. An observational study is another type of clinical research through which researchers collect data and assess health outcomes without changing participants’ care.
Learn more about the clinical trials 澳门正规博彩十大排行平台 is conducting below. If you are interested in learning more about a particular trial, please email medinfo@bmrn.com.
软骨发育不全 is a rare genetic bone growth condition characterized by disproportionate short stature, curvature of the spine and an enlarged head (macrocephaly).
CLN2 (late infantile neuronal ceroid lipofuscinosis type 2) disease is an ultra-rare and rapidly progressing pediatric brain disorder and one of the most common forms of neuronal ceroid lipofuscinosis, a group of inherited disorders collectively known as Batten disease.
杜氏肌营养不良(DMD)是一种隐性疾病, X-linked neuromuscular disorder caused by a severe deficiency or the complete absence of dystrophin, 一种保护肌肉细胞的蛋白质.
A型血友病 is a rare bleeding disorder caused by a mutation in the gene that provides instructions to make the protein FVIII, 什么是血液正常凝结所必需的.
Hypochondroplasia is a rare genetic condition generally characterized by short stature, 矮壮的构建, 胳膊和腿短得不成比例, 广泛的, 手脚短, 轻度关节松弛, 脊柱侧凸, 和巨头.
议员IVA, 也被称为Morquio A, is a rare inherited disease that affects major organ systems in the body. The condition is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder.
粘多糖病(议员六世), 也被称为马罗托-拉米综合征, is an inherited lysosomal storage disorder caused by the deficiency of an enzyme normally required for the breakdown of certain complex carbohydrates.
Phenylketonuria (北大) is an inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH).
A clinical trial is a research study done to evaluate new potential treatments in people. 在临床试验期间, information is collected to determine whether a product candidate is safe and effective, as well as to evaluate the risks and benefits of the medicine. 有关临床试验的更多信息,请访问 临床试验.政府 or The Center for Information and Study on Clinical 研究 Participation
研究 and development occurs in several stages before clinical trials commence in people. Preclinical testing is conducted for each investigational product to evaluate the safety, 功效, 管理的最佳实践, and many other properties before entering human clinical trials. 一旦临床前测试完成, an Investigational New Drug (IND) application is submitted to a regulatory authority, 比如美国.S. 美国食品药品监督管理局, so the regulator can evaluate the safety of the product candidate and ensure that clinical trial participants will not be subjected to an unreasonable risk.
临床试验分四个阶段进行:
研究用药物在人体中进行评估, 通常是在没有这种情况的志愿者身上, for the first time to evaluate its safety and best practices for administering the product candidate.
评估候选产品 to determine a safe dose or range of doses, 进一步评价其安全性, and to begin testing in participants with the condition of interest to determine if it has the intended or predicted effects.
评估候选产品, 通常在持续时间较长的大型试验中, to confirm its effectiveness and to further evaluate safety. Phase 3 trials often compare the study drug to commonly used treatments (if any) or to placebo treatment, if it is scientifically and ethically appropriate to do so.
Post-marketing studies are performed after regulatory agency approval occurs. These studies are designed to collect additional information including the medicine’s risks, 在更广泛的患者群体中的益处和最佳使用, 通常在很长一段时间内. 例如, a Phase 4 study could be a registry of patients with a certain condition to collect medical information and better understand outcomes, both in patients receiving the medicine and those who are not.
1-3期临床试验完成后, a regulatory application is submitted to regulatory agencies. The application contains all data gathered about the safety and effectiveness of the study drug from the preclinical studies and clinical trials that have been performed. The application also contains information about the chemistry, 毒理学, 产品的药理学和制造工艺. 监管机构审查数据,然后, 如果得到批准, the new medicine can be marketed and distributed to the public through prescription by a qualified physician.
在临床试验中测试潜在的新疗法时, it is critical to include the 广泛的est possible range of individuals, to ensure these medicines have the best chance of improving outcomes for the diversity of patients who will ultimately receive them. 在澳门正规博彩十大排行平台, we create pioneering medicines for people with genetic conditions around the world, and we are deeply committed to enrolling representative populations in our clinical trials.