About the International Alliance for Spinal Muscular Atrophy

Mission Statement

To promote international collaborative research efforts, to ensure that there is co-ordinated research and to exchange information.

Agreement

Member countries agree to:

to promote research to find answers for SMA.
to provide information and support families.
to raise public awareness for SMA.
to share pertinent SMA information quarterly or as it becomes known to them with other IASMA members.
to assist in funding research in their country, which has been granted by an IASMA member using the Families of SMA, USA Scientific Advisory Board, or other such IASMA approved scientific board.
to assist with funding in sending representatives to the International SMA Research Group Meeting sponsored annually by Families of SMA, USA.
to share information on research funded by their individual organization and to promote this on our website.
to link their website to the IASMA "common page" allowing access to all IASMA members
to follow basic guidelines, which will be set up by IASMA committee members in regards to information such as carrier frequency, diet, treatments, etc. prior to posting on website.
to follow the International classifications;
- Type I, or Werdnig-Hoffman Disease, is the most severe form of SMA. Children with Type I tend to be weak and lack motor development, rendering movement difficult. Children afflicted with Type I cannot sit unaided and have trouble breathing, sucking and swallowing.
- Type II is slightly less severe. Type II patients may be able to sit unaided or even stand with support and usually do not suffer from feeding and swallowing difficulties. However, they are at increased risk for complications from respiratory infections.
- Type III, also known as Kugelberg-Welander Disease, is the least deadly form of childhood-onset SMA. Type III patients are able to stand, but weakness is prevalent and tends to eventually sentence its victims to a wheelchair. Type III SMA strikes children after the age of 18 months, but can surface even in adulthood.
- Type IV is the adult form of the disease in which symptoms tend to begin after age 35. Symptoms usually begin in the hands, feet and tongue, and spread to other areas of the body.
- Adult Onset X-Linked SMA, also known as Kennedy's Syndrome or Spinal Bulbar Muscular Atrophy, occurs only in men. Facial and tongue muscles are noticeably affected. In addition, these men also often have breast enlargement known as gynecomastia. Like all forms of SMA, the course of the disease is variable, but in general tends to progress slowly.
to promote smn deletion by blood test as the only means to diagnose (except in the case of non-deletion SMA)
to withhold publishing information they have received from IASMA members without their explicit written permission.
to participate in IASMA discussions and to assist in disseminating Information from IASMA members as needed.
to promote the International Patient Registry, held at Indiana University,(USA)
to contribute to an epidemiological study, which will include North America, Europe and countries of those members of the IASMA
to abide by the IASMA confidentiality agreement
to commit to the IASMA mission statement:

To promote international collaborative SMA research efforts and to ensure coordinated research, and to exchange information on SMA.
to use the following slogan in regard to IASMA:

Around the world, working together to make a difference.