Stem-Cell Gamble

June 21, 2011

Stem-Cell Gamble

Technology Review (MIT) | June 21, 2011 | By Antonio Regalado

Hans Keirstead wakes up every morning at his home near Los Angeles and checks CNN. He’s looking for news about the first-ever human test of embryonic stem cells, launched in October by the biotechnology firm Geron. Mostly, he’s looking for bad news. “If someone dies, or is in pain, then it’s over,” he says, pushing a hand through his tawny hair. Keirstead, dressed in a loose linen shirt and wearing a thumb ring, is a biologist at the University of California, Irvine, who has variously been called the “rock star,” “miracle worker,” and “Pied Piper” of stem-cell science. Today he has a corner office in a new $67 million research center paid for in part by California voters, whom he helped persuade to vote for a $3 billion stem-cell spending plan in 2004 with a video of partially paralyzed rats walking again after stem-cell transplants performed in his laboratory.

That same treatment is now being tested in human beings. No wonder Keirstead is anxious. Although he is not directly involved in the clinical trial, the discovery he patented, promoted to Californians, and later licensed to Geron has now become the leading test of whether embryonic stem cells will finally live up to their medical potential. “I’m dying to know if it works,” he says.

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Stem cell study could aid motor neurone disease research

March 1, 2011

Stem cell study could aid motor neurone disease research

Physorg.com | March 1, 2011

Scientists have discovered a new way to generate human motor nerve cells in a development that will help research into motor neurone disease.

A team from the Universities of Edinburgh, Cambridge and Cardiff has created a range of motor neurons – nerves cells that send messages from the brain and spine to other parts of the body – from human embryonic stem cells in the laboratory.

It is the first time that researchers have been able to generate a variety of human motor neurons, which differ in their make-up and display properties depending on where they are located in the spinal cord.

The research, published in the journal Nature Communications, could help scientists better understand motor neurone disease. The process will enable scientists to create different types of motor neurons and study why some are more vulnerable to disease than others.

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Fairfield man shows off his artistic side by painting with his wheelchair in Vacaville

February 21, 2011

Fairfield man shows off his artistic side by painting with his wheelchair in Vacaville

The Reporter | February 21, 2011 | By Melissa Murphy

Doing something he’s always wanted to do, Fairfield resident Jeremy Hatzell will get to paint — with his wheelchair.Using vibrant colors and a canvas spread on the floor, Hatzell used his artistic side to bring the blank sheet to life at the Rhythm in Blue art studio in downtown Vacaville.

“I want it to look like Chuck Norris punched a rainbow,” he said. “I saw a car commercial where a car drove through paint and I thought I could do that too.”

Hatzell wasn’t expected to live past the age of 4 after doctors diagnosed him with spinal muscular atrophy at 8 months old.

At age 33, Hatzell knows there is a reason he is still here.

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New Mouse Models of SMA Developed Using Mouse Smn Genes that Mimic Human SMN2

February 2, 2011

New Mouse Models of SMA Developed Using Mouse Smn Genes that Mimic Human SMN2

FSMA (Press Release) | January 31, 2011

Dr. Christine DiDonato and colleagues at Children’s Memorial Research Center in Chicago, IL  and Ottawa Health Research Institute in Ottawa, ON recently published 2 new mouse models of SMA in the Journal PLoS One with funding from Families of SMA.

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SMA Foundation and Rules-Based Medicine Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

January 19, 2011

SMA Foundation and Rules-Based Medicine Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

Press Release | January 19, 2011

NEW YORK and AUSTIN, Texas, Jan. 19, 2011 /PRNewswire/ — The Spinal Muscular Atrophy (SMA) Foundation and Rules-Based Medicine, Inc. (RBM) announced today that they have reached the first milestone in a program to develop a panel of plasma protein biomarkers for SMA using RBM’s Multi-Analyte Profiling (MAP) technology platform.  In this collaboration, RBM will discover and also confirm plasma protein biomarker candidates previously identified from the multicenter Biomarkers for SMA (BforSMA) clinical study sponsored by the SMA Foundation and conducted by BG Medicine. The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials of SMA therapies.

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Lives ‘will be lost and families torn apart’ if Northern Ireland loses muscle disease advisor

January 17, 2011

Lives ‘will be lost and families torn apart’ if Northern Ireland loses muscle disease advisor

Belfast Telegraph | January 17, 2011 | By Lisa Smyth

A woman with a life-threatening muscle disease has warned lives will be lost if Northern Ireland loses its only specialist care advisor as the NHS struggles to save money.

Northern Ireland will become the only part of the UK without a specialist care advisor for people with degenerative muscle conditions.

Funding runs out for the post at the end of March and so far health bosses have refused to say whether more money will be made available to guarantee the future of the position, which costs the health service just £50,000 a year.

Michaela Hollywood (20) has Spinal Muscular Atrophy and said the specialist care advisor has provided support and assistance during some of the most difficult periods of her life.

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Repligen Receives $1.4 Million Research Grant from the MDA to Support SMA Development Program

December 15, 2010

Repligen Receives $1.4 Million Research Grant from the Muscular Dystrophy Association to Support Spinal Muscular Atrophy Development Program

Repligen Corporation Press Release | December 15, 2010

WALTHAM, Mass., Dec. 15, 2010 /PRNewswire/ — Repligen Corporation (Nasdaq: RGEN) today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association (“MDA”) to support the ongoing development of RG3039 for Spinal Muscular Atrophy (“SMA”).  RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.  The goal of this grant is to support the advancement of RG3039 into human clinical testing, including continued evaluation in preclinical models of SMA, GMP manufacturing for human clinical trials and early clinical evaluation in healthy volunteers and patients.  Funding from the Muscular Dystrophy Association provides important support for Repligen’s development programs and access to a global network of scientists, physicians and patients.  Repligen has previously been awarded two research grants from the MDA to support its Friedreich’s ataxia program.

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Summer and Fall Fundraisers Hosted by SMA Families Raise Over $1.1 Million for Spinal Muscular Atrophy Research and Support!

December 13, 2010

Summer and Fall Fundraisers Hosted by SMA Families Raise Over $1.1 Million for Spinal Muscular Atrophy Research and Support!

FSMA Press Release | December 10, 2010

Since our last update in June, SMA Families have organized 135 fundraising events raising almost $1,170,000 for the important research and critical family support programs that Families of SMA carries out.  The stunning list of events shown below shows the amazing strength in numbers that the SMA community has.

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Carlingford art show to raise research cash

December 10, 2010

Carlingford art show to raise research cash

Hills Shire Times | December 10, 2010 | By Vanessa Bradbury

JENNIFER Burns works as a university relationship manager for the Actuaries Department by day and allows her creative side to reign in her spare time.

This Carlingford visual artist will be hosting an art exhibition at her home on Sunday in a bid to not only showcase her talent but also raise much-needed funds for the Spinal Muscular Atrophy Association of Australia (SMA).

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They’ll make memories, too

December 8, 2010

They’ll make memories, too

The Daily Item | December 8, 2010 | By Evamarie Socha

Toys will not be a problem at the 2010 Wish Kids holiday party in Milton this weekend.

“There are tons of games and so, so much stuff,” party coordinator Kerry Tilford said, “my house looks like Santa’s toy factory.”

The toys take over her Turbotville home until Saturday, when they’ll go to the Make A Wish kids who, with their families, will gather at 1 p.m. at St. Andrew’s Church to have fun and reminisce about their granted wishes.

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