Spinal Muscular Atrophy News and Research RSS Feed - Spinal Muscular Atrophy News and Research

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.
Study on fruit flies, brewer's yeast may provide clues about cause of Spinal Muscular Atrophy

Study on fruit flies, brewer's yeast may provide clues about cause of Spinal Muscular Atrophy

Scientists at the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) have shown that fruit flies and brewer's yeast can reveal clues about the cause of Spinal Muscular Atrophy (SMA), the most common genetic killer of infants. [More]
Innovative gene transfer-based treatment for kids with giant axonal neuropathy

Innovative gene transfer-based treatment for kids with giant axonal neuropathy

University of North Carolina School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). [More]
Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 study in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on children with spinal muscular atrophy (SMA) who have completed the open-label, Phase 2 multiple-dose study of ISIS-SMN Rx and are continuing to receive treatment in an open-label extension (OLE) study. [More]
Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals provides update on ISIS-SMN Rx Phase 2 clinical study in infants with Type I SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMN Rx in infants with Type I spinal muscular atrophy (SMA). [More]
Cytokinetics, The ALS Association expand partnership to fight against ALS

Cytokinetics, The ALS Association expand partnership to fight against ALS

Cytokinetics, Incorporated and The ALS Association announced an expanded partnership in which the company will provide Gold Level Sponsorship of the National Walks to Defeat ALS as well as Platinum Level Sponsorship for ALS Association Golden West Chapter initiatives. [More]
Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Good Start Genetics, Inc., a commercial-stage molecular genetics information company, today presented data from two studies analyzing detection rates among patients at in vitro fertilization (IVF) clinics who underwent carrier screening for inherited diseases. The studies were presented at the 2015 Annual Clinical and Scientific Meeting of The American College of Obstetricians and Gynecologists being held in San Francisco, Calif. [More]
Proteomics helps identify previously unrecognized proteins and pathways in nerve regeneration

Proteomics helps identify previously unrecognized proteins and pathways in nerve regeneration

Using proteomics techniques to study injured optic nerves, researchers at Boston Children's Hospital have identified previously unrecognized proteins and pathways involved in nerve regeneration. Adding back one of these proteins--the oncogene c-myc--they achieved unprecedented optic nerve regeneration in mice when combined with two other known strategies. [More]
Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals announces positive results from ISIS-ANGPTL3Rx Phase 1 study

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-ANGPTL3Rx. In this study, healthy volunteers treated with ISIS-ANGPTL3Rx achieved dose-dependent, statistically significant reductions in angiopoietin-like 3 (ANGPTL3) of up to 93 percent with a mean reduction of up to 84 percent from baseline (p<0.001). [More]
Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE). [More]
Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis receives $5 million from Biogen Idec to develop drugs for neurological disorders

Isis Pharmaceuticals, Inc. announced today it has earned a $5 million milestone payment from Biogen Idec associated with the validation of an undisclosed target to treat a neurological disorder under its broad strategic collaboration with Biogen Idec to develop drugs to treat patients with neurological disorders. [More]
Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis receives $10 million from Biogen Idec to begin IND-supporting studies of ISIS-BIIB4 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec for initiating the investigational new drug (IND)-supporting studies of ISIS-BIIB4 Rx. [More]
Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis announces top-line results from ISIS-PTP1B Rx Phase 2 study in type 2 diabetes patients

Isis Pharmaceuticals, Inc. announced today top-line results from a Phase 2 study of ISIS-PTP1B Rx in patients with type 2 diabetes. In the Phase 2 study patients treated with ISIS-PTP1B Rx achieved statistically significant reductions in body weight and hemoglobin A1c (HbA1c). [More]
AMRI awarded NIH contract for drug development services

AMRI awarded NIH contract for drug development services

AMRI today announced it received a 10-year federal contract award from the National Institutes of Health for drug development and manufacturing services. This NIH/National Institute of Neurological Disorders and Stroke award will support NIH's Drug Manufacturing and Formulation Program (DMFP), which is a component of the Blueprint Neurotherapeutics Network (BPN). [More]
New computational technique reveals genetic roots of autism, cancers

New computational technique reveals genetic roots of autism, cancers

In the decade since the genome was sequenced in 2003, scientists and doctors have struggled to answer an all-consuming question: Which DNA mutations cause disease? [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]
Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Stanley T. Crooke, Ph.D., M.D., founder, CEO and chairman of the board of directors of Isis Pharmaceuticals, Inc., has been awarded the 2014 SCRIP Lifetime Achievement Award, one of the most prestigious of its kind in the pharmaceutical and biotechnology industries worldwide. [More]
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers from the Centre for Comparative Genomics have received almost $800,000 in funding from the National Health and Medical Research Council to develop genetic drugs to treat rare diseases. [More]
Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical studies of ISIS-SMN Rx in infants and children with spinal muscular atrophy at the 19th International World Muscle Society (WMS) Congress in Berlin, Germany. [More]
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