Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Safety Study

May 23, 2011

Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Safety Study

Press Release | May 19, 2011

Families of Spinal Muscular Atrophy announced today that Repligen Corporation has received approval from the U.S. Food and Drug Administration to commence a Phase I safety study in healthy volunteers for RG3039, formerly called Quinazoline495, which is being developed for Spinal Muscular Atrophy.

FSMA began the Quinazoline program in 2000 at the very initial stages of drug development, when risk is the highest. It was the very first industrial drug program for SMA ever conducted. FSMA fully funded the program with investments of $13 Million. The direction from FSMA provided the positive results necessary to license the program to Repligen Corporation and leverage larger funding for clinical development.

This clinical trial will be a double-blind, single ascending dose, Phase I study in healthy, adult volunteers to evaluate the pharmacokinetic and safety profile of RG3039 in up to 40 subjects. The study will be the first step in the clinical development of RG3039 as a potential treatment for SMA.

“It is very exciting after 10 years of hard work by FSMA leading this program, including a significant financial investment, for the drug candidate to be licensed to Repligen in 2009 and to now be able to announce the approval to advance into human clinical trials. This is a tremendous milestone for our community to have reached as we make progress toward a treatment for SMA,” stated Jill Jarecki, Ph.D. Research Director at FSMA. “We are very pleased to have received approval to initiate human clinical trials with RG3039,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation.

Click HERE or on the image below to read more…

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Posted by stronwi

Pranav and the Court Decision: Anti-Stem Cell Lawsuit Still Threatens Sick

May 2, 2011

Pranav and the Court Decision: Anti-Stem Cell Lawsuit Still Threatens Sick

Huffington Post | May 2, 2011 | By Don C. Reed

“He is right here,” said Pranav’s mother, in her lilting Indian accent; “would you like to speak to him?”

For a moment I was actually afraid; I don’t know why.

Pranav had Spinal Muscular Atrophy (SMA) a disease like a slow spinal cord injury. Like the paralysis which afflicts my son Roman Reed, SMA is not “catching”, and in any case we were on the telephone.

“Hello,” said a surprisingly deep voice, very dignified. I had a sudden thought — he sounded like a politician, a good one, somebody who would use the power of government to help people.

We talked about Disneyland and Elmo, both of which he strongly approved.

But the conversation was tiring for him, and we soon said goodbye.

He was three years old.

Click HERE or on the image below to read more…

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FightSMA to host panel discussions on spinal muscular atrophy

April 30, 2011

FightSMA to host panel discussions on spinal muscular atrophy

News Medical | April 30, 2011

International non-profit organization FightSMA will be hosting highly respected experts from across the country on Thursday, May 5, 2011 for a series of panel discussions, Thriving with SMA: LIVE! This panel is a unique, one-of-a-kind series of presentations from influential leaders in the fields of pulmonary issues, orthopedics, nutrition, neurology and clinical trial.

FightSMA will be joined by families, friends, healthcare professionals and more for this historic presentation. In addition, the panel will be offered online with free and unlimited access via the web broadcasting service, UStream. The panel will be live broadcast from 9:00am EDT to 2:00pm EDT on the FightSMA website (www.fightsma.org). Attendees can register for the webcast, as well as submit questions for the panelists by visiting the FightSMA website and pointing towards the “Celebrating 20 Years” block.

“There are hundreds of families across the U.S. and across the globe who simply cannot physically attend the conference for various reasons,” said FightSMA President and founder, Martha Slay. “So, on May 5, we’re bringing the conference to them! Families will be able to ask questions of SMA experts in the areas of nutrition, exercise, pulmonary issues, and clinical trials. This is unprecedented and it’s free, and can be attended from your own computer. Go to www.fightsma.org for more details.”

Click HERE or on the image below to read more…

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Spinal Muscular Atrophy: A Timely Review

April 16, 2011

Spinal Muscular Atrophy: A Timely Review

Archives of Neurology | April 11, 2011 | By Stephen J. Kolb, MD, PhD; John T. Kissel, MD

ABSTRACT

Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by loss of motor neurons in the anterior horn of the spinal cord and resultant weakness. The most common form of SMA, accounting for 95% of cases, is autosomal recessive proximal SMA associated with mutations in the survival of motor neurons (SMN1) gene. Relentless progress during the past 15 years in the understanding of the molecular genetics and pathophysiology of SMA has resulted in a unique opportunity for rational, effective therapeutic trials. The goal of SMA therapy is to increase the expression levels of the SMN protein in the correct cells at the right time. With this target in sight, investigators can now effectively screen potential therapies in vitro, test them in accurate, reliable animal models, move promising agents forward to clinical trials, and accurately diagnose patients at an early or presymptomatic stage of disease. A major challenge for the SMA community will be to prioritize and develop the most promising therapies in an efficient, timely, and safe manner with the guidance of the appropriate regulatory agencies. This review will take a historical perspective to highlight important milestones on the road to developing effective therapies for SMA.

Click HERE or on the image below to read more…

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Trophos Presents Data At The American Academy Of Neurology On SMA And MS

April 15, 2011

Trophos Presents Data At The American Academy Of Neurology On SMA And MS

Medical News Today | April 14, 2011

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today that Trophos has described the design of its pivotal clinical study of olesoxime in spinal muscular atrophy (SMA) via a poster presentation at this week’s 6th Annual American Academy of Neurology (AAN) meeting held in Honolulu, Hawaii, US, April 9 to 16, 2011. In addition at the conference, Trophos also delivered an oral presentation detailing Trophos’ novel approach in multiple sclerosis (MS). Both the paper and oral presentation were delivered at the conference on April 14.

The poster, on the pivotal efficacy study of olesoxime in the rare, neurodegenerative condition, SMA, demonstrates how the challenges of designing a robust study in an indication where there is no precedent were met by bringing together and working with key opinion leaders, patient and physician networks and regulators. The study commenced in October 2010 (see release of October 15 2010) and is currently recruiting patients. Efficacy results are expected in the second half of 2013. The study is substantially funded by Trophos’ partnership with the Association Française contre les Myopathies (AFM) (see release of 19 March 2009) and the trial protocol has benefited from the EMA Protocol Advice procedure.

Click HERE or on the image below to read more…

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Notice of Intent to Publish a Request for Applications for a Spinal Muscular Atrophy (SMA) Biomarker Study (U01) and Convene a Related Workshop

April 13, 2011

Notice of Intent to Publish a Request for Applications for a Spinal Muscular Atrophy (SMA) Biomarker Study (U01) and Convene a Related Workshop

NINDS | April 8, 2011

The National Institute of Neurological Disorders and Stroke (NINDS) intends to promote a new initiative by publishing a Funding Opportunity Announcement (FOA) to solicit applications for clinical research studies that will accelerate and augment the validation of biomarkers for Spinal Muscular Atrophy (SMA).

This Notice is being provided to allow potential applicants sufficient time to develop responsive protocols and necessary collaborations.

The FOA is expected to be published in the NIH Guide for Grants and Contracts in late spring/early summer of 2011, with an anticipated receipt date in late summer of 2011.

The FOA will use the U01 cooperative agreement mechanism. Details of the planned FOA are provided below.

Click HERE or on the image below to read more…

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University of Michigan creates nation’s first embryonic stem cell lines to study blood clotting, neurological disorders

April 4, 2011

University of Michigan creates nation’s first embryonic stem cell lines to study blood clotting, neurological disorders

The Detroit Free Press | April 4, 2011 | By Patricia Anstett

In a development that aims to provide better answers for the treatment of disabling, inherited conditions, the University of Michigan announced today it has created some of the nation’s first embryonic stem cell lines that carry genes responsible for specific diseases.The discovery leapfrogs U-M to a top tier of research institutions. Of nearly 100 embryonic stem cell lines in a national registry, only Harvard and Stanford universities have developed reproducible lines to study specific diseases.

U-M’s research involved the creation of two cell lines for hemophilia B, a clotting disorder, and Charcot-Marie Tooth disease, a neurological disorder that causes weakness in the legs, hands and feet.

“This will fuel the fire of discovery” in how the earliest embryonic cells develop markers of a specific disease, said Gary Smith, Ph.D., co-director of U-M’s Consortium for Stem Cell Therapies and leader of the cell-derivation project there.

U-M expects to develop other cell lines to study other disorders, including Huntington’s disease, myotonic dystrophy, Rett syndrome, spinal muscular atrophy and Tay-Sachs disease.

Click HERE or on the image below to read more…

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New Article In Regenerative Medicine Explains the Potential of Stem Cell Therapies for ALS and SMA

March 19, 2011

New Article In Regenerative Medicine Explains the Potential of Stem Cell Therapies for ALS and SMA

FSMA via Regenerative Medicine | March 16, 2011

Authors Eva Feldman, Nicholas Boulis, and colleagues present supporting evidence for the utilization of stem cell technology in the treatment of amyotrophic lateral sclerosis and spinal muscular atrophy in the Journal Regenerative Medicine.  They also discuss the first-in-human Phase I trial currently underway examining the safety and feasibility of intraspinal stem cell injections in amyotrophic lateral sclerosis patients as a foundation for translating stem cell therapies for various neurological diseases.

Click HERE to read the full article via FSMA in Regenerative Medicine…

Click HERE to read more on the FSMA site…

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Enzo Biochem Unit Launches First-to-Market Survival Motor Neuron (SMN) Protein Immunoassay System

March 16, 2011

Enzo Biochem Unit Launches First-to-Market Survival Motor Neuron (SMN) Protein Immunoassay System

Press Release | March 16, 2011

Enzo Biochem, Inc. and the Spinal Muscular Atrophy (SMA) Foundation today announced that Enzo’s wholly owned subsidiary, Enzo Life Sciences Inc., has launched a unique immunoassay (ELISA) system which can be used for the identification and detection of Survival Motor Neuron (SMN) protein. The kit is the result of a collaborative agreement between Enzo and the SMA Foundation.for the development of reagents and assays for SMN protein. The availability of an effective SMN ELISA could further enable and expedite drug discovery, development and therapy for Spinal Muscular Atrophy, the leading genetic cause of mortality in infants and toddlers.

Click HERE or on the image below to read more…

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Scientists from Genzyme Corporation and ISIS Pharmaceuticals Publish Paper Showing Antisense Oligonucleoties (ASOs) Ameliorate Symptoms in a Severe Mouse Model of Spinal Muscular Atrophy

March 4, 2011

Scientists from Genzyme Corporation and ISIS  Pharmaceuticals Publish Paper Showing Antisense Oligonucleoties (ASOs) Ameliorate Symptoms in a Severe Mouse Model of Spinal Muscular Atrophy

Science Translational Medicine (via FSMA) | March 4, 2011

It was reported in the journal Science Translational Medicine that ASOs designed to correct splicing of the SMN2 gene improve symptoms in a severe mouse model of Spinal Muscular Atrophy when delivered directly to the CNS.

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to treat SMA is to use antisense oligonucleotides (ASOs) to redirect the splicing of the SMN2 gene to boost production of functional SMN.

Click HERE or on the image below to read more…

4 Comments | Research, United States | Tagged: , | Permalink
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