We’re very excited to announce that the Consensus Statement for Standard of Care in Spinal Muscular Atrophy has been published in the August 2007 supplement of the Journal of Child Neurology. The recommendations in the document were prepared by an an international body of clinical experts. The panel worked with more than 60 spinal muscular atrophy experts in the field to achieve consensus on 5 care areas: diagnostic/new interventions, pulmonary, gastrointestinal/nutrition, orthopedics/rehabilitation, and palliative care.

To read the document, download it here in PDF form.

From the SMA Project:

Dear Colleague,

We have now posted the Supporting Documentation (including the Subcontract Agreement) for the SMA Project (http://www.smaproject.org) Request for Proposals (RFP) LD-090707. This RFP seeks to identify facilities to conduct preclinical testing of chemical compounds with the ultimate goal of submitting an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). We have also posted an updated version of the RFP (Attachment B in the RFP has been updated).

The RFP and Supporting Documentation can be accessed through the Solicitations page of the SMA Project website at http://www.smaproject.org/solicitations.html. Proposals are due by 3 p.m. Eastern Time on October 5, 2007.

Studies to be conducted under the resulting contract will include preclinical safety, toxicology, pharmacokinetic/dynamic, and biodistribution studies with candidate small molecule compounds that act as therapeutic agents for spinal muscular atrophy (SMA). While a broad range of Good Laboratory Practice (GLP) and non-GLP studies is anticipated, capability to conduct studies according to GLP is required. Science Applications International Corporation (SAIC), on behalf of the Government, shall supply the Offeror with a small amount of each candidate therapeutic compound. For certain compounds provided to the Contractor, SAIC may request a comprehensive Product Development Plan for services to be provided by the Contractor. Facilities will be evaluated on their past experience in preclinical, IND-directed small molecule development, as well as the proposed schedule and cost of studies required.

The National Institute for Neurological Disorders and Stroke (NINDS) (http://www.ninds.nih.gov/) launched The SMA Project: A Collaborative Program to Accelerate Therapeutics Development for Spinal Muscular Atrophy in September 2003. This program funds milestone-driven research aimed at identifying and rapidly developing a treatment for SMA, a paralyzing neurodegenerative disease of childhood.