Sentences with phrase «spinal muscular atrophy»

The Boston startup said its deal with the New York - based Spinal Muscular Atrophy Foundation calls for two years of research and development funding from the foundation.

Spinraza, the first Food and Drug Administration - approved treatment for spinal muscular atrophy, had quarterly...

Healthy mice will rear about six times, mice with spinal muscular atrophy treated with both therapies reared about four times and mice with either drug alone only reared about two times.

«This is the first study done that identifies a target, JNK3, that's independent of the genetic mutation of spinal muscular atrophy for novel therapeutic development.»

Leg muscle function and fatigue during walking in spinal muscular atrophy type 3.

«We don't know yet if the SMN lnc - RNA actually plays a role in the disease, but it is a convenient target that we've used to our advantage to treat spinal muscular atrophy in mice,» says Sumner.

This year, for example, scientists in Germany and at the health institutes showed that valproic acid, an epilepsy drug, could more than double S.M.N. protein levels in cell cultures taken from spinal muscular atrophy patients.

We cloned the gene for neuromuscular degeneration in a mouse model for a lethal infantile form of SMA known as spinal muscular atrophy with respiratory distress.

Cameron, now 3 years old, almost died as a baby from spinal muscular atrophy but a new drug appears to have preserved the motor neurons that control his muscles.

«New line of attack on spinal muscular atrophy.»

Prospective cohort study of spinal muscular atrophy types 2 and 3.

Lagoon also plans to continue raising money for spinal muscular atrophy research, which he's been doing since his daughter's birth.

The closest approximation to another RNAi success comes from Ionis Pharmaceuticals and Biogen, which last year received approval for Spinraza, a drug aimed at spinal muscular atrophy.

Last year Ionis had a massive success with an ASO for another brain disease called spinal muscular atrophy (SMA).

New York, NY — September 24, 2004 — Researchers at Columbia University, Children's Hospital Boston and The Children's Hospital of Philadelphia have established a clinical research network to study spinal muscular atrophy (SMA), the leading genetic killer of infants and toddlers.

April 24 - Drugmaker Biogen Inc's quarterly revenue missed analysts» estimates on Tuesday as it had disappointing sales of its promising drug Spinraza to treat spinal muscular atrophy because fewer new patients used the expensive treatment.

Congratulations to Umrao Monani, Assistant Professor at the Motor Neuron Center of Columbia University and Cathleen Lutz, Associate Director of Genetic Research Science at The Jackson Laboratory, and their colleagues for their new publication «Postsymptomatic Restoration of SMN Rescues the Disease Phenotype in a Mouse Model of Severe Spinal Muscular Atrophy» in the Journal of -LSB-...]

Kittens can develop Spinal Muscular Atrophy, a condition that causes paralysis in the rear legs.

Only sufficient federal dollars now stand between spinal muscular atrophy and a cure.

Spinal muscular atrophy funding peaked this year at $ 5 million, after remaining under $ 3 million for several years.

PROVIDENCE, R.I. [Brown University]-- Scientists and doctors know that the devastating disease spinal muscular atrophy (SMA) arises from a problem with both copies of the SMN1 gene, leading to a lack of the survival motor neuron (SMN) protein.

Spinal muscular atrophy involves wasting of the skeletal muscles and is caused by gradual degeneration of nerve cells in the spinal cord.

Nusinersen and similar compounds being developed for spinal muscular atrophy focus on increasing or making more of the SMN gene product that gets converted into usable protein.

«Candidate drug provides benefit in spinal muscular atrophy animal models.»

Though spinal muscular atrophy (SMA) in its most severe form remains incurable and fatal in early childhood, researchers are sustaining a multipronged counterattack for patients and their families.

Amyotrophic lateral sclerosis (ALS) affects adults while spinal muscular atrophy (SMA) mostly strikes young children.

«Drug for spinal muscular atrophy prompts ethical dilemmas, bioethicists say.»

«Researchers seek biomarker to assess spinal muscular atrophy treatment.»

People with spinal muscular atrophy lack a protein that is required for the motor nerve cells in the spinal cord to function.

In a recent study published in the December 15th issue of Human Molecular Genetics, Dr. Gangwani and his team of researchers at TTUHSC El Paso describe how mice with spinal muscular atrophy saw great improvement when the JNK3 enzyme was genetically inhibited to eliminate its activity.

Ataxia Congenital muscular dystrophy Congenital myasthenic syndrome Fronto - temporal lobe dementia Hereditary motor neuropathies — Charcot - Marie - Tooth disease Hereditary spastic paraplegias Huntington's disease Muscular channelopathy Muscular dystrophy Spinal muscular atrophy — Lower motor neuron disease

The SMA Foundation has worked in partnership with several other Spinal Muscular Atrophy organizations to establish national and international networks of clinics ready for clinical trials.

From a clinical perspective, Jessell's findings have paved the way for the treatment of neurodegenerative diseases, notably spinal muscular atrophy and Lou Gehrig's disease.

He was a post-doctoral fellow at the Children's Hospital of Eastern Ontario in Ottawa, working on identification of genes associated with human spinal muscular atrophy.

She learns that both she and her husband are carriers for the recessive lethal childhood disorder spinal muscular atrophy, and they seek further counseling.

Genetic circuitry of Survival motor neuron, the gene underlying spinal muscular atrophy Proc Natl Acad Sci U S A. 2013 Jun 11; Authors: Sen A, Dimlich DN, Guruharsha KG, Kankel MW, Hori K, Yokokura T, Brachat S, Richardson D, Loureiro J, Sivasankaran R, Curtis D, Davidow LS, Rubin LL, Hart AC, Van Vactor D, Artavanis - Tsakonas S

2013 May 16; Authors: Kissel JT, Elsheikh B, King WM, Freimer M, Scott CB, Kolb S, Reyna SP, Crawford TO, Simard LR, Krosschell KJ, Acsadi G, Schroth MK, D'Anjou G, Lasalle B, Prior TW, Sorenson S, Maczulski JA, Swoboda KJ, For the Project Cure Spinal Muscular Atrophy Investigators» Network

The National Institute of Neurological Disease and Stroke recently designated spinal muscular atrophy as the model for its new approach to funding «translational research.»

Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analyses.

About SMA Spinal Muscular Atrophy (SMA) is a rare, and often fatal, genetic disorder that affects approximately 1 in every 10,000 births.

Hereditary spinal muscular atrophy: an autosomal dominant degenerative disease of motor neurons characterized by weakness and muscle atrophy with a typical gait, and progressing to dangling of the head and a drooping, paralyzed tail.

a. Spinal muscular atrophy; b. Multiple sclerosis; c. Spinal stenosis; d. Locked - in syndrome; e. Severe conversion disorder; f. Traumatic spinal injury; g. Parkinson's disease; and h. Huntingdon's disease

If you would prefer to make a donation via check (with no minimum), please send to the following address: Spinal Muscular Atrophy Foundation, 888 Seventh Ave., Suite 400, New York, NY 10019.