Thanks to the efforts of the SMA Coalition and Helping Hands, strong FY06 Report Language was included in both the House and Senate Labor, Health and Human Services, and Education Appropriations Committee Reports, urging increased focus on SMA:

HOUSE FY06 Labor, Health and Human Services and Education Appropriations -

NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

Spinal Muscular Atrophy- SMA is the leading genetic killer of infants and toddlers. The Committee understands that the severity of the disease, its relatively high incidence, and the possibility of imminent treatments have led NINDS to initiate the SMA Therapeutics Development Program. The Committee commends NINDS for this initiative and encourages the Institute to continue to commit the resources to ensure a timely completion of the project to identify and complete preclinical research and development of candidate therapeutics for treating SMA by 2007. To maximize program efficiency, it is also important that NINDS lead efforts to integrate Therapeutics Development efforts with emerging programs in the biotech and pharmaceuticals industry, academic medical centers and collaborations with voluntary health organizations to ensure that duplication of effort is avoided.  The Committee understands that the strategy for developing a treatment for SMA will guide therapeutics development for other diseases including: Duchenne Muscular Dystrophy, ALS, Huntington's and Alzheimer's.

NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT

Spinal Muscular Atrophy [SMA]- The Committee recognizes the efforts at NINDS as the leading institute against SMA in recent years, and strongly encourages other institutes, notably NICHD, to expeditiously work collaboratively to support and strengthen the SMA Project.  The Committee fully expects that NICHD will explore new potential avenues to collaborate with NINDS, given the fact that SMA is the leading genetic killer of infants and toddlers.

NIH OFFICE OF THE DIRECTOR

Spinal Muscular Atrophy- The Committee encourages of the Office of the Director to ensure support of the SMA Project by providing active and ongoing support from the OD as well as from other related Institute Directors, particularly NICHD, since SMA is the leading genetic killer of infants and toddlers.

SENATE FY06 Labor, Health and Human Services and Education Appropriations-

NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

Spinal Muscular Atrophy. - SMA is the leading genetic killer of infants and toddlers. The Committee understands that the severity of the disease, its relatively high incidence, and the possibility of imminent treatments have led NINDS to initiate the SMA Therapeutics Development Program. The Committee commends NINDS for this initiative and strongly urges NIH/NINDS to continue to commit the resources to ensure a timely completion of the project mission -- to identify and complete preclinical research and development of candidate therapeutics for treating SMA by 2007. To maximize program efficiency, it is also critical that NINDS lead efforts to integrate Therapeutics Development efforts with emerging programs in the biotech and pharmaceuticals industry, academic medical centers and collaborations with voluntary health organizations to ensure that duplication of effort is avoided.  The Committee encourages NINDS to aggressively expand its SMA basic, translational and clinical research portfolio. The Committee understands that the strategy for developing a treatment for SMA will guide therapeutics development for other diseases including: Duchenne Muscular Dystrophy, ALS, Huntington's and Alzheimer's. The Committee strongly urges NINDS to successfully and expeditiously execute the SMA Therapeutics Development Program for the benefit of patients of SMA and countless other diseases. The Committee requests that NIH report back to the Committee, no later than June 1, 2006 with a progress report on all aspects of SMA research.  

NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT

Spinal Muscular Atrophy [SMA]- SMA is the leading genetic killer of infants and toddlers, and is the most prevalent genetic motor neuron disease. The severity of the disease, its relatively high incidence, and the possibility of imminent treatments have led NINDS to initiate the SMA Project. The Committee believes that the treatment of SMA, and the SMA Project at NINDS, is strategically consistent with the mission of the NICHD, with the NIH Roadmap initiative and its specific emphasis on cross-institute research integration. The Committee is concerned that, to date, the NICHD has not made any progress toward working collaboratively with NINDS to support and expand the SMA Project. The Committee strongly urges NICHD to do so expeditiously and to report back to the Committee no later than May 1, 2006.

OFFICE OF THE DIRECTOR

Spinal Muscular Atrophy- The Committee strongly urges the OD to ensure the success of the SMA Project by providing active and ongoing support from the OD as well as from other related Institute Directors. While the Committee commends the active work and progress of NINDS on the SMA Project, the Committee remains concerned that other Institutes, to date, has made no commitment to SMA research.  The Committee reiterates its request of last year that the OD take all necessary steps to ensure that relevant Institutes are fully engaged by devoting attention and resources to SMA and specifically to the SMA Project.