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Home > SMA Research > Spinal Muscular Atropy Research News > 2009

2009 Research News

December 21, 2009	California Stem Cell Closes Financing Round to Enable Advancement to Human Clinical Trials Including for Spinal Muscular Atrophy. Click here for the press release.
December 17, 2009	Families of Spinal Muscular Atrophy Research Funding Leads to the Publication of 18 Journal Articles in 2009. Please click here to learn more about this research.
December 11, 2009	Science-Business Exchange Publishes Cover Story on Families of Spinal Muscular Atrophy Funded Paratek Drug Program for SMA Click here to see the article.
December 8, 2009	Stem Cell Based Treatment For Spinal Muscular Atrophy Receives Orphan Drug Designation. California Stem Cell, Inc And Families Of Spinal Muscular Atrophy Reach Significant Milestone In Development Of Motor Neuron Treatment For SMA.
December 3, 2009	Isis Pharmaceuticals Adds A Spinal Muscular Atrophy Drug to Development Pipeline. Please click here to read the press release about ISIS-SMNRx to treat SMA.
December 1, 2009	Enrollment Complete in the VALIANT Clinical Trial of Type III Spinal Muscular Atrophy Patients Funded by Families of SMA. Families of SMA and the Project Cure SMA clinical trial network are pleased to announce that the Phase II VALIANT SMA clinical trial led by Dr. John Kissel at The Ohio State University has completed enrollment.
November 10, 2009	Families of Spinal Muscular Atrophy Funded Study Shows Quinazoline Compounds Give Survival Benefit in a Mouse Model of SMA. Lead author Dr. Matthew Butchbach at the Ohio State University publishes SMA paper in the Journal Human Molecular Genetics.
November 4, 2009	Paratek Pharmaceuticals, Cold Spring Harbor Lab, Rosalind Franklin Researchers Publish on Tetracycline Drug Candidate for Spinal Muscular Atrophy. First publication from the drug discovery collaboration at Paratek Pharmaceuticals that has been funded by Families of SMA.
October 22, 2009	Families of Spinal Muscular Atrophy Licenses Potential Treatment for SMA to Repligen Corporation. Click here for the full press release.
October 15, 2009	Paratek Pharmaceuticals Inc. and Families of Spinal Muscular Atrophy Announce a Multi-Million Dollar Award. From the National Institute of Neurological Diseases and Stroke to Advance a Drug Candidate for Spinal Muscular Atrophy.
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.
September 28, 2009	Multi-Center Trial of Valproic Acid and Carnitine in Infants with Type I Spinal Muscular Atrophy Has Completed Enrollment. The Project Cure SMA clinical trial group has completed enrollment of 36 infants with Type I SMA into the CARNI-VAL Type I Trial.
September 11, 2009	Spinal Muscular Atrophy Researcher Named Director of NINDS Office of Clinical Research Dr. Petra Kaufmann will now direct clinical research at NINDS.
August 28, 2009	Families of Spinal Muscular Atrophy Receives FDA Orphan Drug Designation For Quinazoline495 For The Treatment of Spinal Muscular Atrophy. First Time a Novel Therapy Specifically Designed For SMA Has Ever Been Awarded Orphan Drug Status by the FDA.
August 19, 2009	Geron's Plans for First Clinical Trial for Spinal Cord Injury with Stem Cell Therapy Placed on Hold.
August 1, 2009	Highlights from 2009 International Spinal Muscular Atrophy Research Group Meeting
July 27, 2009	Genzyme Genetics Publishes a Paper Showing Carrier Rates for Spinal Muscular Atrophy The paper published in Journal Of Medical Genetics shows differing carrier rates among ethnic groups in North America.
July 16, 2009	New Research Publications from Families of Spinal Muscular Atrophy Funded Grants. Three new publications were recently released that received funding from the Families of SMA basic research program.
July 16, 2009	The Laboratory of Dr. Chistina Brahe Publishes a SMA Paper in the European Journal of Human Genetics In a paper partially funded by FSMA, lead author Dr. F.D. Tiziano publishes a paper entitled, SMN transcript levels in leukocytes of SMA patients determined by absolute real-time PCR".
July 14, 2009	FSMA Funded Lead Author, Dr. Kum-Loong Boon from the Laboratory of Dr. Christine Beattie, Publishes a Paper in Human Molecular Genetics The paper shows data that a zebrafish model of SMA has motor neuron defects at the neuromuscular junction. Please click above to read more details about this work.
July 14, 2009	New Paper Published in Human Molecular Genetics from the Laboratory of Dr. Brunhilde Wirth In the paper partially funded by Families of SMA, the authors present data showing the HDAC inhibitor LBH589, a drug currently being developed for cancer, increases SMN protein levels up to 10 times. Please click above to read more details about the paper.
June 18, 2009	Latest Developments on Three Drug Discovery Programs Funded by Families of Spinal Muscular Atrophy.
June 2, 2009	Sadly, Dr. Hong Li, a SMA researcher at Institute of Molecular Biology at Academia Sinica In Twain, passed way on March 7th, 2009. He will be long remembered in the SMA community for his seminal contribution of developing one of the first animal models of SMA. Please click here to see his obituary.
May 13, 2009	New Paper Published on Spinal Muscular Atrophy Clinical Trial. Project Cure SMA Group Publishes Open Label Valproic Acid Trial Results Funded by Families of SMA.
April 28, 2009	Families of Spinal Muscular Atrophy; University of California, Irvine Stem Cell Scientist; and California Stem Cell, Inc. Extend Collaboration to Progress Stem Cell Therapy for Spinal Muscular Atrophy to Clinical Trials.
April 21, 2009	Families of Spinal Muscular Atrophy Awards $400,000 to Support Ongoing Clinical Trials Read more here.
April 9, 2009	Families of Spinal Muscular Atrophy Announces New Clinical Trial Site for SMA At Duke University in North Carolina.
March 19, 2009	Trophos to Perform Efficacy Trial of Olesoxime in Spinal Muscular Atrophy. Read the details here.
March 9, 2009	President Barack Obama Signs Stem Cell Executive Order. Relevance to Spinal Muscular Atrophy Research.
March 6, 2009	Families of Spinal Muscular Atrophy Announces 6 New Basic Research Awards for 2009. New Edition of Families of Spinal Muscular Atrophy Research Publication “Compass” Available Now.
March 2, 2009	New Video from Families of Spinal Muscular Atrophy Funded Researcher Dr. Hans Keirstead The challenges and opportunities of developing stem cell therapy for SMA.
January 23, 2009	Geron Receives FDA Clearance to Begin World's First Human Clinical Trial of Embryonic Stem Cell-Based Therapy for Spinal Cord Injury. This will help pave the regulatory path for Spinal Muscular Atrophy.
January 22, 2009	New Paper on Spinal Muscular Atrophy Published in the Journal of Neuroscience By Dr. Charlotte Sumner and colleagues.
January 16, 2009	American College of Medical Genetics Issues Guidelines on Carrier Screening for Spinal Muscular Atrophy By Dr. Thomas Prior of Ohio State University.