cells that responded to the experimental treatment.

The company also tested AT2220 in 72 healthy volunteers and found it to be safe and well tolerated at all doses studied.

In November, Amicus announced it plans to further develop AT2220 in partnership with Shire Human Genetic Therapies, part of the multinational Shire biopharmaceutical company ( www.shire.com).

MDA, SMA groups release
family version of SMA
Standard of Care

The Patient Advisory Group of the International Coordinating Committee (ICC) for SMA Clinical Trials has published a family-friendly set of guidelines for care in spinal muscular atrophy (SMA) to complement the physician guidelines published in August. (See Research Updates, November-December 2007.)

The ICC includes representatives of MDA, the SMA Foundation, FightSMA and Families of SMA.

The new publication, “A Family Guide to the Consensus Statement for Standard of Care in Spinal Muscular Atrophy,” is designed to guide patients and families in their discussions with doctors and health-care specialists. The committee emphasizes that these guidelines are only suggestions and should not be considered absolute requirements for care.

The Guide has recommendations on confirming the diagnosis; managing breathing, eating and nutrition, movement and daily activities; and preparing for illness.

The entire document can be read and printed out on the MDA Web site at www.mda.org/publications/family_ guide_sma_standard_of_care.pdf.

MDA joins SMA groups in
support of SMA Treatment
Acceleration Act

MDA has joined the SMA Foundation, Families of SMA, and FightSMA in endorsing the SMA Treatment Acceleration Act (H.R. 3334/S.2042). The Act, if passed

by Congress, will aid investigators in conducting national clinical trials to identify treatments for spinal muscular atrophy (SMA). It would provide federal support to complement the substantial private funding that national nonprofit organizations, including MDA, are now providing.

GINA gets thumbs-up
from Congress

Under the provisions of legislation approved by Congress this spring, insurance companies now are prohibited from requiring people to undergo genetic tests, and from denying them health care insurance or increasing their premiums based on genetic test results.

The Genetic Information Nondiscrimination Act (GINA) also prohibits employers from using genetic test results when making hiring, compensation, assignment or promotion decisions.

President George Bush signed the act on May 21. q