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Post date: May 17, 2013 2:31:33 PM
December 27, 2009
H.R.2149 SMA Treatment Acceleration Act
Update on current Sponsor and cosponsors of the SMA Treatment Acceleration Act in the House of Representative. The bill is still waiting in the Community of Energy and Commerce committee there has been no action reported to date. Please view list and we encourage you if you do not see your State Representative listed, to contact them and ask them to cosponsor this bill. Thank you.
Sponsor:
Cosponsor:
1. Donna Christensen [D-VI] (new)
4. Tammy Baldwin [D-WI2] (new)
6. Dan Boren [D-OK2] (new)
7. Frederick Boucher [D-VA9] (new)
8. Bruce Braley [D-IA1] (new)
9. George Butterfield [D-NC1] (new)
12. Christopher Carney [D-PA10 (new)
13. Kathy Castor [D-FL11] (new)
15. Jim Cooper [D-TN5] (new)
20. Jeffrey Fortenberry [R-NE1] (new)
23. Barton Gordon [D-TN6] (new)
24. Raymond Green [D-TX29] (new)
26. Ralph Hall [R-TX4] (new)
28. Martin Heinrich [D-NM1] (new)
29. Stephanie Herseth Sandlin [D-SD]
35. Steve Kagen [D-WI8] (new)
36. Dale Kildee [D-MI5] (new)
37. Ronald Kind [D-WI3] (new)
43. Michael McCaul [R-TX10] (new)
44. Thaddeus McCotter [R-MI11]
46. Cathy McMorris Rodgers [R-WA5]
48. James Moran [D-VA8] (new)
49. Glenn Nye [D-VA2] (new)
50. Collin Peterson [D-MN7] (new)
51. Ted Poe [R-TX2] (new)
55. Ileana Ros-Lehtinen [R-FL18] (new)
57. Bobby Rush [D-IL1] (new)
60. Louise Slaughter [D-NY28] (new)
61. Fortney Stark [D-CA13] (new)
65. Debbie Wasserman Schultz [D-FL20]
66. Lynn Westmoreland [R-GA3] (new)
68. Charles Wilson [D-OH6] (new)
71. David Wu [D-OR1]
S. 1158 SMA Treatment Acceleration Act
Update on current Sponsor and cosponsors of the SMA Treatment Acceleration Act in the Senate. This bill has been read by the Committee on Health, Education, Labor, and Pensions, but to date we have nothing to report. Please view list and we encourage you if you do not see your State Representative listed, to contact them and ask them to cosponsor this bill. Thank you.
Sponsor:
Sen. Debbie Ann Stabenow [D-MI]
Cosponsors:
11. John Reed [D-RI]
December 8, 2009
STEM CELL BASED TREATMENT FOR SPINAL MUSCULAR ATROPHY RECEIVES ORPHAN DRUG DESIGNATION
STEM CELL BASED TREATMENT FOR SPINAL MUSCULAR ATROPHY RECEIVES ORPHAN DRUG DESIGNATION
IRVINE, Calif. (December 7, 2009)
California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that the FDA has granted orphan drug designation to MOTORGRAFTTM, a stem cell-derived motor neuron product, for the treatment of Spinal Muscular Atrophy (SMA).
Orphan drug designation, granted by the FDA Office of Orphan Products Development, provides several incentives to companies in the private sector developing novel drugs or biologics to treat diseases with relatively small market potential. These include seven years market exclusivity following FDA approval, clinical trial design assistance, reduced user fees and tax credits.
CSC developed MOTORGRAFT™ as a stem cell-derived motor neuron replacement product for the treatment of SMA. Pre-clinical GLP safety and efficacy studies, funded significantly by the Families of Spinal Muscular Atrophy and conducted by Professor Hans S. Keirstead of the University of California at Irvine, have demonstrated safety and functional benefit in several animal models.
November 9, 2009
Researchers identify drug candidate for treating Spinal Muscular Atrophy
Cold Spring Harbor, N.Y. – A chemical cousin of the common antibiotic tetracycline might be useful in treating spinal muscular atrophy (SMA), a currently incurable disease that is the leading genetic cause of death in infants. This is the finding of a research collaboration involving Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory (CSHL) and scientists from Paratek Pharmaceuticals and Rosalind Franklin University of Medicine and Science.
The new molecule boosts the levels of SMN protein in cells by fixing a mistake in a cellular processing mechanism called RNA splicing. In a study that will appear in the journal Science Translational Medicine on November 4th, the scientists report this fix in both mouse models of SMA, as well as in cells isolated from SMA patients.
Unlike previously identified molecules that stimulate SMN production, the tetracycline-like compound is a unique therapeutic candidate in that it is a small molecule that specifically alters RNA splicing by directly targeting the splicing reaction.
October 26, 2009
ISU researchers find possible treatment for Spinal Muscular Atrophy
ISU researchers find possible treatment for Spinal Muscular Atrophy
Spinal Muscular Atrophy is the second-leading cause of infant mortality in the world. Ravindra Singh, associate professor in biomedical sciences at Iowa State University's College of Veterinary Medicine, would like to see Spinal Muscular Atrophy lose its high ranking and even slide off the list altogether.
Most Spinal Muscular Atrophy sufferers -- more than 95 percent -- have a mutated or deleted gene called Survival Motor Neuron 1 (SMN1) that doesn't correctly do its job of creating functional SMN proteins.
Singh's solution is to replace that poor-performing gene with another gene.
Humans need a certain level of SMN protein to ward off Spinal Muscular Atrophy.
When SMN1 fails to create functioning proteins, Spinal Muscular Atrophy is the result.
There is a gene already in humans that looks very much like SMN1, so much so that it's called SMN2. The SMN2 gene doesn't seem to serve any function that researchers can identify.
Singh has discovered a way of using SMN2 to produce the working SMN protein. When SMN2 makes enough SMN, it compensates for the mutated or malfunctioning SMN1 gene.
All proteins in human bodies are made by copying genes. This copy is called pre-mRNA.
Pre-mRNA then becomes mRNA by splicing out certain parts of the sequence that are non-coding, meaning they don't help the function of the gene.
These non-coding portions of the pre-mRNA are called intronic sequences, sometimes referred to as junk sequence because it is originally copied from junk DNA.
SMN2 normally doesn't produce normal protein because of the presence of a specific intronic sequence in the gene or DNA.
To make SMN2 behave as SMN1, Singh has introduced a small antisense oligonucleotide that blocks this specific intronic sequence.
When the intronic sequence is blocked, SMN2 produces normal proteins and acts, in effect, like SMN1.
"The significance of our work is that we have this stuff called junk DNA in SMN2," said Singh. "We found that we could get SNM2 to behave as SMN1 by introducing a small oligonucleotide. It is a very simple experiment if you think about it."
The resulting proteins are normal just like a regular cell - free from Spinal Muscular Atrophy. Read More
October 14, 2009
California Stem Cell, Inc. And Families Of Spinal Muscular Atrophy Announce Completion Of Pre-Ind Meeting With FDA For Stem Cell-Derived Therapy For The Treatment Of Spinal Muscular Atrophy Type I
IRVINE, Calif. (October 14, 2009)
California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that they have completed a formal pre-Investigational New Drug (Pre-IND) meeting with the Food and Drug Administration (FDA) for guidance on the clinical and regulatory pathway and requirements for submission of an IND to initiate human trials for a stem cell-derived motor neuron replacement therapy for Spinal Muscular Atrophy (SMA) Type I.
SMA is the leading genetic cause of death of infants. It is a disorder that results from a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing and breathing.
October 2, 2009
2009 H1N1 Flu and Spinal Muscular Atrophy.
September 30, 2009.
Information for SMA Patients and Families.
What is influenza?
Influenza (the flu) is an illness caused by a virus. It usually happens in the fall and winter but people can get the flu at other times of the year. The flu is easily spread by direct contact, coughing, sneezing, and when an infected person touches a surface that others then use, like doorknobs and railings.
What is 2009 H1N1 flu?
2009 H1N1 (referred to as “swine flu” early on) is a new influenza virus causing illness in people. This new virus was first detected in people in the United States in April 2009. This virus is spreading from person-to-person worldwide, probably in much the same way that regular seasonal influenza viruses spread. However, it has the potential to cause more severe disease than seasonal influenza especially in individuals with SMA. Click here to read more
September 30, 2009
H.R.2149 SMA Treatment Acceleration Act
Update on current Sponsor and cosponsors of the SMA Treatment Acceleration Act in the House of Representative. The bill is still waiting in the Community of Energy and Commerce committee there has been no action reported to date. Please view list and we encourage you if you do not see your State Representative listed, to contact them and ask them to cosponsor this bill. Thank you.
S. 1158 SMA Treatment Acceleration Act
Update on current Sponsor and cosponsors of the SMA Treatment Acceleration Act in the Senate. This bill has been read by the Committee on Health, Education, Labor, and Pensions, but to date we have nothing to report. Please view list and we encourage you if you do not see your State Representative listed, to contact them and ask them to cosponsor this bill. Thank you.
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