Thanks to the efforts of the SMA Coalition and Helping Hands, strong FY05 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 FY05 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 is pleased that initial work has begun on the program and encourages NIH/NINDS to move forward with the mission to develop a treatment for SMA ready for clinical trials within four years. The Committee further urges NINDS to develop a strategy for executing effective clinical trials for future therapies. The Committee encourages NINDS to integrate Therapeutics Development efforts with:  the biotech and pharmaceuticals industry, academic medical centers and collaborations with voluntary health organizations.

NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT

Spinal Muscular Atrophy.-- Spinal Muscular Atrophy [SMA] is the leading genetic killer of infants and toddlers and is the most prevalent genetic motor neuron disease. Over 25,000 Americans, mostly children, suffer from significant physical disability and impairment as a result of SMA. The Committee encourages NICHD to work closely with NINDS to develop collaborations which will support the SMA Therapeutics Development project-- including an expansion of the scope and level of SMA research at NICHD. In addition, NICHD is strongly urged to develop formal programs that increase public and professional awareness of SMA.

SENATE FY05 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 is pleased that initial work has begun on the program and strongly urges NIH/NINDS to commit the resources required to ensure a timely completion of the project mission--a treatment for SMA ready for clinical trails for current and future therapies. To maximize program efficiency, it is also critical that NINDS integrate Therapeutics Development efforts with the biotech and pharmaceuticals industry, academic medical centers and collaborations with voluntary health organizations. 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 April 2005 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 the NICHD. The Committee strongly urges the NICHD to work closely with NINDS to develop collaborations and programs which will support and expand the SMA Project. The Committee strongly urges NICHD to expand the scope and level of SMA research by aggressively soliciting grant applications on an expedited basis. Lastly, the Committee strongly urges the NICHD to develop formal programs that increase public and professional awareness of SMA. The Committee requests that the NICHD report back to the Committee during the fiscal year 2006 appropriations hearings.

NIH 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, most notably NICHD. The OD is urged to take all necessary steps to ensure that the NICHD is fully engaged by expanding their scope and level of resources dedicated to SMA.