Spinal Muscular Atrophy News and Research

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Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.

SMA researchers receive grant for Spinal Muscular Atrophy research

Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from Sophia's Cure Foundation via the Pepsi Refresh Project.

9 Sep 2010

iPierian raises $28 million from Series B equity financing closure

iPierian, Inc., the leading biopharmaceutical company using patient-derived induced pluripotent stem cells (iPSCs) for drug discovery, today announced the second closing of its Series B equity financing, bringing the total amount raised in this round to $28 million.

9 Sep 2010

Research on motor neuron subtype may provide useful therapy for neuron diseases

Scientists have devised a method for coaxing mouse embryonic stem cells into forming a highly specific motor neuron subtype. The research, published by Cell Press in the September 3rd issue of the journal Cell Stem Cell, provides new insight into motor neuron differentiation and may prove useful for devising and testing future therapies for motor neuron diseases.

3 Sep 2010

Study finds genetic change in chromosome 4 causes FSHD

The Muscular Dystrophy Association today heralds a landmark muscular dystrophy advance by an international study team of scientists and physicians from the Netherlands, United States, France and Spain. Led by MDA-grantee Silvère van der Maarel, Ph.D., at Leiden University Medical Center in the Netherlands, the collaborative study of more than 2,300 people found that two distinct genetic changes on chromosome 4 must be present to cause facioscapulohumeral muscular dystrophy (FSHD).

21 Aug 2010

Muscular Dystrophy Association awards $14.1M in new grants

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

20 Aug 2010

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

18 Aug 2010

Dr. Hastings receives grant for Spinal Muscular Atrophy research

Michelle Hastings, PhD, Assistant Professor in the Department of Cell Biology and Anatomy, in the Chicago Medical School of Rosalind Franklin University of Medicine and Science, has been awarded a five-year $1.68 million grant from the National Institutes of Health to support her research aimed at investigating treatments for the fatal children's disease, Spinal Muscular Atrophy (SMA).

11 Aug 2010

Spinal muscular atrophy may lead to cardiac dysfunction: Study

Along with skeletal muscles, it may be important to monitor heart function in patients with spinal muscular atrophy (SMA). These are the findings from a study conducted by Nationwide Children's Hospital and published online ahead of print in Human Molecular Genetics. This is the first study to report cardiac dysfunction in mouse models of SMA.

11 Aug 2010

Repligen total revenue for first-quarter fiscal 2011 increases 39% to $7.01 million

Repligen Corporation today reported results for the first quarter of fiscal year 2011, ended June 30, 2010. Total revenue for the first quarter was $7,010,000 compared to total revenue of $5,061,000 for the first quarter of fiscal year 2010, an increase of $1,949,000 or 39%. Bioprocessing product revenue for the first quarter was $4,269,000 compared to $2,473,000 for the first quarter of fiscal 2010, an increase of $1,796,000 or 73%.

5 Aug 2010

Long-term exposure to valproate leads to bone loss: Study

Scientists are reporting a possible explanation for the bone loss that may occur following long-term use of a medicine widely used to treat epilepsy, bipolar disorder, and other conditions. The drug, valproate, appears to reduce the formation of two key proteins important for bone strength, they said. Their study, which offers a solution to a long-standing mystery, appears in ACS' monthly Journal of Proteome Research.

5 Aug 2010

Braun Corporation presents key themes, recommendations discussed at symposium for the disabled

The Braun Corporation, the world's largest manufacturer of wheelchair-accessible vans, ramps and wheelchair lifts, today published key insights from a symposium hosted recently in Washington, D.C. titled "ADA 20/20: Looking Back, Looking Forward on Mobility." In anticipation of the 20th anniversary of the Americans with Disabilities Act, the panel of key thought leaders from government, medicine, academia and business exchanged insights on current thinking around future mobility needs for Americans with disabilities.

27 Jul 2010

Today's OpEds: Health reform and long-term care; Health care incentives; Reasons for optimism or repeal and replace?

Our system of long-term care does not provide adequate or coordinated human services and medical treatment for our growing aging population. … Thanks to a provision of the new health care law known as the Community Living Assistance Services and Supports program, tomorrow's continuum of care does not have to be so unforgiving. For the first time, a voluntary public insurance program will be available to help adults and families pay for long-term care services (Bruce Chernof, 7/23).

26 Jul 2010

CSHL scientists reverse symptoms of Type III SMA using ASOs

The devastating, currently incurable motor-neuron disease spinal muscular atrophy might soon be treated with tiny, chemically modified pieces of RNA called antisense oligonucleotides.

13 Jul 2010

iPierian announces closing of $22M Series B equity financing round

iPierian, Inc., the leading biopharmaceutical company using patient-derived induced pluripotent stem cells (iPSCs) for drug discovery, today announced the closing of a $22 million Series B equity financing. The financing round was led by new investor Google Ventures, and was joined by Mitsubishi UFJ Capital and ATEL Ventures. All current investors also participated, including: Kleiner Perkins Caufield & Byers, Highland Capital Partners, MPM Capital and FinTech Global Capital.

9 Jul 2010

University of Utah makes progress against SMA with support of Families of Spinal Muscular Atrophy

18 Jun 2010

Researchers make significant headway in fight against SMA

Researchers at the University of Utah are making strides in understanding and combating a motor neuron disease known as Spinal Muscular Atrophy. The spinal muscular atrophies are a group of inherited diseases that cause progressive degeneration of motor nerves resulting in muscle weakness. The most common form of the disorder, due to the deletion of a critical gene on chromosome 5, is currently the leading cause of inherited infant and child mortality.

18 Jun 2010

Novel gene replacement therapy holds promise for Spinal Muscular Atrophy

Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.

10 Jun 2010

Repligen's fiscal 2010 total revenue decreases to $20,971,000

Repligen Corporation today reported results for the fourth quarter and fiscal year 2010, ended March 31, 2010. Total revenue for the year was $20,971,000 compared to total revenue of $29,362,000 for fiscal year 2009 ended March 31, 2009.

9 Jun 2010

Ohio scientists demonstrate existence of effective technology for nationwide newborn screening of SMA

Scientists in Ohio studying Spinal Muscular Atrophy (SMA) have concluded that the technology now exists to carry out nationwide screening of newborn children and pregnant mothers. The study, published in the American Journal of Medical Genetics, reveals that effective screening may allow parents to find proactive treatments before the symptoms become irreversible.

7 May 2010

CML, FightSMA Canada collaborate to find a cure for spinal muscular atrophy

CML HealthCare Income Fund (TSX: CLC.UN), (CML) of Mississauga, Ontario has partnered with FightSMA Canada, to help find a cure for Spinal Muscular Atrophy (SMA), the leading genetic cause of death among children under the age of two.

3 May 2010