澳门葡京赌场注册基因疗法 is dedicated to developing innovative therapies for patients suffering from rare and life-threatening neurological genetic diseases. Before these therapies can be approved and commercially 可用 to patients, they must undergo clinical trials to evaluate their safety and effectiveness.

While it is generally preferred that patients gain access to investigational therapies by participating in clinical trials, patients may not be eligible or able to take part in these studies. 病人 with serious or life-threatening diseases or conditions sometimes seek therapies that are not yet approved or 可用 in their country. The 澳门葡京赌场注册基因疗法 "管理访问程序" addresses this need by making certain investigational or unapproved therapies 可用 to eligible patients.

临床试验

Before an investigational therapy can be placed on the market, it must undergo well-controlled clinical trials to evaluate its safety and effectiveness, as well understand whether its potential benefit to patients outweighs the possible risks.

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管理访问程序

The 澳门葡京赌场注册基因疗法 global 管理访问程序 (MAP) is designed to provide a potential pathway for patients seeking such a treatment, provided the required eligibility criteria are met and in accordance with applicable local laws and regulations.

The 澳门葡京赌场注册基因疗法 “Managed 访问” terminology covers all locally defined pre-approval access mechanisms and programs such as “Compassionate Use”, “扩展访问”, “命名病人供应”, “特别接达计划/计划”, 和其他人.

Current 澳门葡京赌场注册基因疗法 “管理访问程序”:

  • Spinal muscular atrophy (SMA) 管理访问程序.

This global 管理访问程序 is 可用 to eligible patients with spinal muscular atrophy (SMA) in countries w在这里 Zolgensma® (onasemnogene abeparvovec) treatment has not received regulatory approval or formal access approval. 对于这个全球地图, the physician will need to assess patient eligibility by determining whether the patient meets pre-defined medical and program-specific criteria.

参与全球MAP, patients must meet all eligibility criteria outlined in the treatment plan, 包括但不限于:

  • 病人 under the age of two with genetically confirmed bi-allelic mutations in the SMN1 gene; regardless of type, 拷贝数, 或者症状发作.
  • 在地理上, the patient must be a citizen or legal resident of, 有资格享受医疗保健, a country w在这里 Zolgensma has not received regulatory approval or formal access approval.
  • Implementation of the program will need to comply with the specific legal and regulatory framework that applies in every country.

Medical eligibility needs to be confirmed and Health Authority approval obtained in the country w在这里 the patient will be treated. In situations w在这里 the number of requests outweigh 可用 supply, a third-party administers a blinded allocation. If 澳门葡京赌场注册基因疗法 is not able to provide our treatment to the patient at that time, the patient automatically rolls over to the pool for the next allocation as long as he/she remains medically eligible and it is still permissible in the country.

Our one-time gene therapy is provided free-of-charge. To ensure safe administration and adequate follow-up, treatment can only be administered in trained treatment centers. If no such center exists in the patient’s country of residence, 澳门葡京赌场注册基因疗法 determines the feasibility of alternative treatment center locations.

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Who should I contact for more information?

用于一般医疗信息查询, healthcare professionals may submit requests to the 澳门葡京赌场注册基因疗法 Medical Information team in the US, 欧洲，中东和亚洲, 拉丁美洲 & 加拿大, or 亚太地区.

How do I submit a request for Managed 访问?

A request must be submitted by the treating physician on behalf of the patient. Requests can be submitted via 澳门葡京赌场注册基因疗法 request portal 在这里. 还有使用门户的指南 可用.

病人/parents/caregivers should contact their treating physician and ask him/her about this global MAP.