ETX boy raises money for SMARD research

September 27, 2011

ETX boy raises money for SMARD research

KLTV | September 23, 2011 | By Lauren Callahan

WHITE OAK, TX (KLTV) – An East Texas boy has had Spinal Muscular Atrophy Respiratory Distress, or “SMARD”, since he was three months old.

And until recently, no one was researching the disease.

Now that a laboratory in Maine is devoted to learning more about “SMARD,” three-year-old Dakin Lovelace wants to help.

Dakin Lovelace was a healthy baby, according to his mom Devon. But when he was three-months-old, he stopped breathing. After many doctor’s visits, he was diagnosed with SMARD

“And so as a result of that Dakin can’t breathe on his own, he can’t walk. He can’t sit up on his own. Um, we are very fortunate, though, that he’s so strong. We’re very, very lucky, and he’s happy. His brain is not affected at all. He’s smart as a whip and he’s a happy boy,” says Devon.

His parents resigned themselves to the fact that Dakin would always be in a wheelchair and using a ventilator. Until they learned this summer about the Jackson Laboratory in Maine, where a researcher is starting to study SMARD.

“We lived pretty much three years having no hope whatsoever that anything anybody was doing was going to help Dakin,” Devon expressed.

There are about 60 known cases of SMARD worldwide.

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Cedars-Sinai Opens New Induced Pluripotent Stem Cell Core Production Facility

September 22, 2011

Cedars-Sinai Opens New Induced Pluripotent Stem Cell Core Production Facility

Press Release | September 21, 2011

Newswise — LOS ANGELES – Sept. 21, 2011 – The Cedars-Sinai Regenerative Medicine Institute has opened a new Induced Pluripotent Stem Cell (iPSC) Core Facility to produce powerful cells capable of making all tissues of the body from adult human skin cells.

One of the first to open in California, cells produced by the Cedars-Sinai core will be used in research funded by the National Institutes of Health and the California Institute for Regenerative Medicine. The cells will be critical for innovative research aimed at increasing our understanding of human diseases and genetic disorders, and the quest for new treatments.

“The opening of the Cedars-Sinai Stem Cell Core Facility underscores what an exciting time this is in regenerative medicine,” said Shlomo Melmed, MD, senior vice president of academic affairs and dean of the faculty at Cedars-Sinai. “It also is an example of Cedars-Sinai’s deep commitment to the scientific research that will be translated into tomorrow’s leading-edge treatments.”

The new facility will use the latest technology to generate induced pluripotent stem cells from a patient skin scraping. The induced pluripotent stem cells can be replicated indefinitely and have biological properties similar to embryonic stem cells. These “blank slate” cells can then be turned into any kind of differentiated cell, such as a brain cell or an eye cell or a liver cell.

Although iPS cells were first produced only three years ago, they have quickly become valuable research tools. Clinicians can take skin cells from patients with specific life threatening diseases. Then, Regenerative Medicine Institute scientists can create iPS cells from them and then generate so-called “disease in a dish” models that enable them to more easily identify effective therapies.

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The Story and Purpose Behind Zane’s Run

September 22, 2011

The Story and Purpose Behind Zane’s Run

Malvern Patch | September 20, 2011 | By Pete Kennedy

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In 2009, Keith and Hillary Schmid lost their 5-month-old daughter, Zane, to type I Spinal Muscular Atrophy, a genetic disease that did not affect Zane’s twin sister Avery.

That year, the Schmids organized Zane’s Run, a 5K held at Sugartown Elementary School. The proceeds of the run helped supply informational packets and car seats to families facing the disease. The third annual Zane’s Run will take place Sept. 25 this year.

View the attached video to learn more about the Schmids’ story.

The Schmids hope to raise awareness and encourage people to be tested for SMA.

“Both parents have to be carriers of the disease to have a child with SMA,” Hillary Schmid said. “[Two carriers] have a 25 percent chance of having a child with SMA, a 50 percent chance of having a child be a carrier, and the other 25 percent chance that the child will be unaffected.”

Click HERE or on the image below to read more…

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New Class of Stem Cell-Like Cells Discovered in Spinal Cord Offers Possibilities for Spinal Cord Repair

September 15, 2011

New Class of Stem Cell-Like Cells Discovered in Spinal Cord Offers Possibilities for Spinal Cord Repair

Press Release | September 15, 2011

SEATTLE, Sept. 15, 2011 /PRNewswire/ — The Allen Institute for Brain Science announced today the discovery of a new class of cells in the spinal cord that act like neural stem cells, offering a fresh avenue in the search for therapies to treat spinal cord injury and disease. The published collaborative study, authored by scientists from the University of British Columbia, the Allen Institute for Brain Science and The Montreal Neurological Institute and Hospital at McGill University and titled “Adult Spinal Cord Radial Glia Display a Unique Progenitor Phenotype,” appears in the open access journal PLoS One.

The research team utilized the Allen Spinal Cord Atlas, a finely detailed genome-wide map of gene expression throughout the mouse spinal cord, to compare the genes expressed, or turned on, in adult spinal cord radial glia with those found in other neural stem cells, revealing a signature set of 122 genes that indicate the likeness of these cells to classic neural stem cells.

The nervous system has historically been thought to be incapable of repairing itself, as the cells used to create it are exhausted during development. With the identification of these new stem cell-like radial glial cells, it may be possible to activate a certain set of genes in order to encourage those cells to reconstruct a damaged network in the adult spinal cord.

“By using the Allen Spinal Cord Atlas, we were able to discover a brand new cell type that has previously been overlooked and that could be an important player in all manner of spinal cord injury and disease, including multiple sclerosis and ALS,” said Jane Roskams, Ph.D., neuroscientist at the University of British Columbia and senior author of the study.

From disabled veterans to those afflicted with Lou Gehrig’s disease (ALS) or Spinal Muscular Atrophy, spinal cord related diseases and disorders affect people of all ages including nearly one-quarter of a million Americans who have suffered from a spinal cord injury; as many as 30,000 Americans who suffer from ALS at any given time; and approximately 2.5 million people worldwide who suffer from multiple sclerosis.

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Trophos completes patient enrolment in pivotal efficacy study of olesoxime in Spinal Muscular Atrophy

September 8, 2011

Trophos completes patient enrolment in pivotal efficacy study of olesoxime in Spinal Muscular Atrophy

Press Release | September 8, 2011

Marseille France, September 8, 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, announced today the completion of patient enrolment in the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA). Over 160 patients have been recruited into the trial since its initiation in October 2010. The study is substantially funded by Trophos’ partnership with the Association Française contre les Myopathies (AFM) (see release of 19 March 2009). The trial protocol has benefited from the EMA protocol advice procedure. Efficacy results are expected in the second half of 2013.

“The completion of recruitment in this pivotal clinical study in only ten months given the rarity of SMA is a great achievement and a major step in the development of olesoxime as a potential treatment for SMA,” said Jean-Louis Abitbol, chief medical officer at Trophos. “SMA is a debilitating and disabling neuromuscular disease and there is a critical need for a treatment that can slow down or prevent the loss of muscle function in SMA patients, for whom no specific treatment exists today. Over 160 patients have been included in the study in only ten months, which reflects both the great commitment of patients and clinicians to find a treatment for SMA and the motivation and hard work of all involved. We anticipate the results of the trial in the second half of 2013 and hope this will be a historic moment for the medical community as well as those affected now and in the future.”

“Thanks to the donations to the French telethon, we have been supporting the development of olesoxime since the first screening up to and including the ongoing clinical phases. The recruitment in this important clinical study has just been completed with great efficiency and brings hope for a first potential treatment to SMA patients,” said Christian Cottet, CEO, AFM.

“Trophos and the AFM been working together for over a decade and this crucial clinical study with Trophos’ olesoxime in SMA is the fruit of our long standing partnership,” said Damian Marron, CEO, Trophos. ”Olesoxime has a promising profile as a potential treatment for SMA and we are hopeful the results of this study will demonstrate that promise, bringing a much needed treatment option and new hope to SMA patients and their families. This study as well as our soon to be completed pivotal study of olesoxime in amyotrophic lateral sclerosis (ALS) underlines Trophos’ commitment to developing breakthrough therapies for rare and serious neurodegenerative diseases.”

Click HERE or on the image below to read more…

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Mogul Using $100 Million in Race to Cure Daughter Lures Novartis

September 8, 2011

Mogul Using $100 Million in Race to Cure Daughter Lures Novartis

San Francisco Chronicle | September 8, 2011 | By Robert Langreth and Alex Nussbaum

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Goldman Sachs Group Inc. (GS) partner Dinakar Singh discovered in 2001 that his 19-month-old daughter, Arya, had a crippling genetic disease called spinal muscular atrophy.

The malady makes the nerve cells that control muscles gradually deteriorate. There are no treatments, let alone a cure, Bloomberg Markets magazine reports in its October issue. Worse still, while the gene causing the ailment had recently been discovered, nobody in the drug industry was doing much about it, he says.

“I was fearful and anxious that treatments would be developed, but far too late to save Arya,” says Singh, 42, who founded and runs New York hedge fund TPG-Axon Capital Management LP, which has $8.1 billion in assets. “We didn’t want to find out 25 years later that the science was really there but there isn’t a drug because nobody focused on it.”

Singh, who left Goldman in 2004, has spent almost $100 million of his own money to create and fund the Spinal Muscular Atrophy Foundation. He wants to discover and develop a drug that he hopes will help his daughter, who is one of 25,000 SMA patients in the U.S. Children with severe forms often die within a few years, while those with mild cases can live a normal life span with supportive care. Arya, 11, and starting sixth grade, uses a wheelchair.

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2 Comments | Advocacy, New York, Research, SMA Foundation, United States | Tagged: , , , , | Permalink
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Ipierian Hires New CEO to Carry on With Stem Cells for Discovering Neurological Drugs

September 8, 2011

Ipierian Hires New CEO to Carry on With Stem Cells for Discovering Neurological Drugs

xconomy | September 7, 2011 | By Luke Timmerman

iPierian, the South San Francisco-based developer of stem cell technology co-founded by top scientists at Harvard University, said today it has hired a new CEO. Nancy Stagliano, the co-founder and former CEO of South San Francisco-based CytomX Therapeutics, has taken the top job at iPierian, while interim CEO Peter Van Vlasselaer has moved aside to become the executive chairman of the board.

The company has been quiet since May, when Xconomy broke the news that the iPierian’s board terminated CEO Mike Venuti and most of the senior management team. A few weeks later, board chairman Corey Goodman resigned in the wake of the company’s shift in strategy. Today’s statement from iPierian says that Van Vlasselaer, the interim CEO, has prioritized the company’s efforts over the past several months toward using its stem cell technology to help discover drugs for neurodegenerative disorders like Alzheimer’s, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy.

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In Summer’s memory… SMA Awareness Month in Narooma

September 8, 2011

In Summer’s memory… SMA Awareness Month in Narooma

Narooma News | September 7, 2011 | By Stan Gorton

SPINAL Muscular Atrophy (SMA) Awareness Month was a massive success in Narooma and honoured the memory of the beautiful Summer Carol Lynn Hayes.
This is according to Summer’s mother Tamara Hayes, who is driven by the memory of her daughter who died from SMA earlier this year.

There is still time to help out by booking a table at the upcoming charity night at the Narooma Golf Club as there are plenty of spaces available.

The former Narooma resident now works for the Spinal Muscular Atrophy Association of Australia in Canberra.

Proof of campaign’s success is her belief that Narooma and surrounds are now aware that SMA is the number 1 genetic killer of infants under the age of 2.

One in 35 of us carry the gene, and if two people carrying this gene together have a baby there is a one in four chance that the baby will suffer from SMA.

And 80 per cent of babies with SMA have Type 1, for which there is no cure and means they will not live to see their second birthday.

Babies born with Type 2 or 3, will need around the clock assistance for their whole lives and life expectancy will be reduced due to age of symptoms onset.

Awareness of SMA was given a boost in Canberra recently when Steve Doszpot, a local Liberal, gave a speech in the Legislative Assembly highlighting the effects of SMA on both children, and the families and friends who support them.

“He acknowledged the tragedy that had befallen my family when we lost Summer, earlier this year and he offered to help with any future fundraising activities and awareness programs that would be held in order to further spread an understanding of this killer disease throughout the community,” she said.

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‘Madison’s Place’ vows to put smiles on many faces

September 2, 2011

‘Madison’s Place’ vows to put smiles on many faces

Woodbury Bulletin | September 2, 2011 | By Rihem Feshir

Woodbury resident Dana Millington wants her children to remember their sister, Madison Claire, in a happy sense. To be able to smile when they hear her name.

Though still grieving after losing their baby girl at age of 2 from complications of spinal muscular atrophy (SMA), the Millingtons knew they wanted to do something to help children with disabilities.

They wanted to bring “Madison’s Place” to the east metro.

The all-inclusive playground will be designed with a special, wheelchair-accessible, rubber surface for children with physical hardships who cannot otherwise enjoy summers at the park. Plans call for the playground to be built at Bielenberg Sports Center.

Fundraising for the project began in 2008 and since then, the Madison Claire Foundation raised $60,000. A September gala is aiming to collect another $100,000.

“I hope it’ll be full of smiles and busy,” Millington said of the playground.

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Digital painter uses one finger to create artwork

September 2, 2011

Digital painter uses one finger to create artwork

Sun Sentinel | September 2, 2011 | By Jonathan D. Marcus

A computer-based painter who uses one finger to create colorful artwork recently exhibited his work in Delray Beach.

A.J. Brockman’s six pieces at the “Vivid Expression in Art” exhibition at the 110 East Atlantic Avenue gallery presented the creative possibilities of painting without traditional brushes and paint.

Brockman, a 23-year-old Palm Beach Gardens resident, has spinal muscular atrophy. The disease, which is a form of muscular dystrophy, is progressive and characterized by the absence of a protein responsible for making muscle.

“A.J. is a well-respected young man with a positive attitude. He lives life as it is,” said gallery arts curator Grace Greenberg.

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