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.

A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 Jan 2010

CSC completes a Series B financing round

California Stem Cell, Inc. (CSC), a leading stem cell company, announced that it has completed a Series B financing round with private investors. The financing will enable the company to support the advancement of its therapeutic products to human clinical trials, as well as the generation and distribution of its tools to aid in stem cell research and drug discovery.

21 Dec 2009

CSC's stem cell-derived motor neuron product for SMA receives FDA orphan drug designation

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that the FDA has granted orphan drug designation to MOTORGRAFT™, a stem cell-derived motor neuron product, for the treatment of Spinal Muscular Atrophy (SMA).

9 Dec 2009

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

Isis Pharmaceuticals, Inc. announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.

4 Dec 2009

Second-quarter fiscal 2010 results announced by Repligen

Repligen Corporation today reported results for the second quarter of fiscal year 2010, ended September 30, 2009. Total revenue for the quarter was $5,421,000 compared to total revenue of $5,090,000 for the second quarter of fiscal year 2009 ended September 30, 2008.

5 Nov 2009

Tetracycline-like compound raises hope for treatment of spinal muscular atrophy

A chemical cousin of the common antibiotic tetracycline might be useful in treating spinal muscular atrophy (SMA), a currently incurable disease that is the leading genetic cause of death in infants. This is the finding of a research collaboration involving Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory (CSHL) and scientists from Paratek Pharmaceuticals and Rosalind Franklin University of Medicine and Science.

5 Nov 2009

Families of Spinal Muscular Atrophy and Repligen partner to develop a treatment for SMA

Families of Spinal Muscular Atrophy (FSMA) announced today that it has entered into a groundbreaking exclusive license agreement with Repligen Corporation for the development of a potential treatment of Spinal Muscular Atrophy (SMA).

23 Oct 2009

Intellectual property covering compounds that have utility in treating SMA licensed

Repligen Corporation announced today that it has entered into an exclusive license agreement with Families of Spinal Muscular Atrophy (FSMA) for intellectual property covering compounds which may have utility in treating Spinal Muscular Atrophy (SMA).

23 Oct 2009

iPierian to participate in $3.7 million NIH grant awarded to The Johns Hopkins University

iPierian, Inc., a biopharmaceutical company focused on changing the paradigm of drug discovery and development through the application of cellular reprogramming, today announced that it has been selected to participate in a $3.7 million National Institutes of Health (NIH) Grand Opportunities (GO) grant awarded to The Johns Hopkins University.

22 Oct 2009

Multi-million dollar cooperative agreement for SMA drug development program

Paratek Pharmaceuticals (Boston, MA) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announced today that a jointly funded drug development program for Spinal Muscular Atrophy (SMA) has been awarded a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS).

19 Oct 2009

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.

9 Sep 2009

Psychogenics and Astrazeneca sign agreement to identify new compounds for treating Central Nervous System disorders

PsychoGenics Inc. and AstraZeneca have entered into a drug discovery and development agreement to identify compounds that are likely to be useful for the treatment of certain Central Nervous System (CNS) disorders.

31 Aug 2009

Office of Orphan Products Development of the FDA grants Orphan Drug Designation to Quinazoline495

Families of Spinal Muscular Atrophy (Libertyville, IL) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted Orphan Drug Designation to Quinazoline495 for the treatment of Spinal Muscular Atrophy.

29 Aug 2009

Discovery of potential treatment for spinal muscular atrophy

Spinal Muscular Atrophy is the second-leading cause of infant mortality in the world. Ravindra Singh, associate professor in biomedical sciences at Iowa State University's College of Veterinary Medicine, would like to see Spinal Muscular Atrophy lose its high ranking and even slide off the list altogether.

28 Jul 2009

Rare window on spinal muscular atrophy genetics

Caused by a mutation of the SMN gene, spinal muscular atrophy (SMA) is an infantile and juvenile neurodegenerative disorder where motor neuron loss causes progressive paralysis.

6 Apr 2009

Scientists make electrically active motor neurons from iPS cells

Stem cells scientists at UCLA showed for the first time that human induced pluripotent stem (iPS) cells can be differentiated into electrically active motor neurons, a discovery that may aid in studying and treating neurological disorders.

25 Feb 2009

Discovery of target that could ease spinal muscular atrophy symptoms

There is no cure for spinal muscular atrophy (SMA), a genetic disorder that causes the weakening of muscles and is the leading genetic cause of infant death, but University of Missouri researchers have discovered a new therapeutic target that improves deteriorating skeletal muscle tissue caused by SMA.

7 Jan 2009

Patient-derived induced stem cells retain disease traits

The dying cells - the same type lost in patients with the devastating neurological disease spinal muscular atrophy - confirmed that the University of Wisconsin-Madison stem cell biologist had recreated the hallmarks of a genetic disorder in the lab, using stem cells derived from a patient.

22 Dec 2008

Trans-splicing therapy for spinal muscular atrophy closer to clinical use

Spinal muscular atrophy, a neurodegenerative disorder that causes the weakening of muscles, is the leading cause of infant death and occurs in 1 in 6,000 live births.

16 Dec 2008

Carrier screening for spinal muscular atrophy should be offered to all couples

Carrier screening for spinal muscular atrophy (SMA) - a serious genetic disease affecting approximately 1 in 10,000 infants that causes progressive muscle weakness and death - should be made available to all families, according to a new practice guideline issued by the American College of Medical Genetics (ACMG).

10 Dec 2008