Sentences with phrase «sma drug»
In vitro assays available for SMA drug development.
http://www.smafoundation.org/
Last spring Dinakar and Loren decided to conduct an investment - banker - type road show to drum up interest among small drug companies, complete with PowerPoint slides estimating potential annual sales for an SMA drug ($ 250 million to $ 750 million) and possible spinoff applications for other diseases.
«The goal of the deCODE collaboration was to generate a final optimized compound with the required properties of an effective SMA drug, called a «Clinical Candidate».
We are very optimistic about the potential of moving this drug to IND within the next year, and the lessons we are learning will help strengthen the SMA drug pipeline.»
Ionis» SMA drug was subsequently approved in the US, EU and many other countries, and is now being given to children with SMA around the world.
The market potential for an SMA drug could exceed $ 500 million, Curis said.
In their news release, the EveryLife Foundation for Rare Diseases highlights a key aspect of this legislation as pertinent to SMA drug development — it will «empower [the] FDA to use all the science -LSB-...]
Neurology Today: SMA Drug Development Gains Momentum Under NINDS Model for Neuro - Research Partnerships.
The U.S. Food and Drug Administration approved the SMA drug, nusinersen, later that year.
The goal of early SMA drug discovery programs has been to identify small molecules that induce the SMN gene to produce sufficient levels of protein to improve motor neuron functioning in affected patients.
Curis, a firm in Cambridge, Massachusetts, has accepted a three - year, $ 5.4 million grant from the SMA Foundation to use its unique technology for growing motor neurons in test tubes to identify potential SMA drugs.
Not exact matches
Now, testing of that compound is leading to a better prognosis for mice with the disease and the possibility of potential drugs that will improve outcomes for patients with SMA.
Overall, this SMA candidate drug is an exciting time as we take another step forward towards developing therapies for SMA patients.»
But early approval left medical centers scrambling to establish treatment programs, which became more urgent when the FDA approved the drug for all SMA patients in December 2016, Burgart said.
Burgart said it's an exciting time for treating spinal muscular atrophy, or SMA, and that the drug is a «game - changer» for families.
Nusinersen, which is injected into the spine and works by temporarily enabling SMA patients to make more of the survival motor neuron protein, is one of the most expensive drugs on the market.
Although there is no cure for SMA currently, the National Institutes of Health (NIH) has listed SMA as the neurological disease closest to finding a cure, due in part to effective drugs like the one developed in Lorson's lab.
Oligonucleotide treatments recently have been approved by the Food and Drug Administration for two neuromuscular diseases: Duchenne's muscular dystrophy and spinal muscular atrophy (SMA).
To receive nusinersen, the babies had to have developed SMA symptoms between 3 weeks and 6 months of age; the drug was injected into the cerebrospinal fluid surrounding the spinal cord.
Dr. De Vivo emphasized that in developing drugs for orphan diseases such as SMA, collaborative programs are a must, and that «strong relationships with disease advocacy foundations are absolutely key.»
«We are committed to quickly developing this drug and are finalizing what we believe will be a rapid development path for this drug in all types of SMA.
New York, NY and Waltham, MA — September 18, 2007 — The Spinal Muscular Atrophy Foundation and BG Medicine today announced a collaboration to discover plasma biomarkers of drug efficacy for spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers.
Lexicon has also entered into an agreement with the SMA Foundation for the potential development of drugs based on discoveries resulting from the program.
The Woodlands, Texas — May 11, 2006 — Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that it was awarded a grant from the United States Army Medical Research & Materiel Command (USAMRMC) for the identification of targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA), a neurodegenerative disorder and the leading genetic cause of death in early childhood.
The Phase 1 study of ISIS - SMNRx is a single - dose, dose - escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2 - 14 who are medically stable.
Which is why Cure SMA Canada and the Canadian Organization for Rare Disorders are calling upon all Canadians to sign this petition, demanding governments include all SMA patients in the funding of Spinraza and to make the drug available immediately.
Current development - stage programs include antisense drugs to treat SMA, ISIS - SMNRx, and myotonic dystrophy type 1, ISIS - DMPKRx.
The hopeful news, Dr. Pacifici said, is that «a lot of these orphan diseases are ripe for drug discovery, especially SMA and Huntington disease.»
«We are very pleased to see the great milestone of a disease - modifying drug treatment advancing into clinical trials in SMA patients,» said Kenneth Hobby, President of Families of SMA.
They join a distinguished group of business and scientific leaders who help formulate and execute the SMA Foundation's drug discovery programs.
SOUTH PLAINFIELD, NJ — December 6, 2007 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small - molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation.
«We see real promise in therapeutic strategies for SMA that increase production of the SMN protein,» said Muscular Dystrophy Association Executive Vice President Research and Medical Director Valerie Cwik, M.D. «We're delighted ISIS Pharmaceuticals is moving forward with a Phase 1 dose - escalation study of its antisense drug in children with SMA.»
This year's ASENT meeting featured a symposium on spinal muscular atrophy (SMA), an inherited motor neuron disease that is the second most common autosomal recessive disorder of children and the most common genetic cause of death in infancy, as a case study in neurology orphan drug development.
Families of SMA applauds ISIS for investing in and leading drug developments efforts for this devastating, orphan disease.»
This marks a welcome new stage of advancement in SMA therapeutics development — and a clear demonstration of the value of non-profit drug development activities.
Congratulations to SMA Foundation Board Member, Stephen Mikita, Esq., on his new role as a Patient Representative to the U.S. Food and Drug Administration (FDA).
The United States Food and Drug Administration granted Orphan Drug Designation with Fast Track Status to ISIS - SMNRx for the treatment of patients with SMA.
About the SMA Foundation Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment for Spinal Muscular Atrophy through targeted funding of clinical research and novel drug development efforts.
The SMA Foundation has diverse drug discovery collaborations with both academia and the private sector to accelerate the development of a treatment or cure.
Once we evaluate ISIS - SMNRx as a single - dose in children with SMA, we will move to multiple - doses in our Phase 1 studies and eventually evaluate the drug in Phase 2 studies in children with SMA, including infants with Type I SMA.»
Speakers said that drug development for muscle - wasting SMA offers promise not only for those with this disease, but also possibly for patients with other diseases such as amyotrophic lateral sclerosis (ALS), muscular dystrophy, and Parkinson disease.
ANN ARBOR, Mich. — Sept. 15, 2008 — Assay Designs and the Spinal Muscular Atrophy Foundation (SMAF) are very pleased to announce a collaborative agreement for development of reagents and assays for SMN (Survival Motor Neuron) protein to expedite drug discovery and development efforts for spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers.
SMA is a rare disorder and could be eligible for orphan status by regulatory authorities, thereby potentially reducing the time needed for a drug to reach patients.
The next step will be to try to cure the SMA in the dish by replacing defective genes or screening for an effective drug.
The SMA Foundation maintains an in vivo drug testing pipeline, where compounds are screened for their efficacy using a standardized platform in a model of severe SMA, the Delta7 mouse, and more recently in the C / C mouse that models milder forms of SMA.
Please see the SMA Foundation report on our mouse drug testing pipeline, for a summary of results and available resources.
While TSA is expensive to produce and it is not approved for clinical use, similar drugs being developed to treat cancer and other diseases may be useful for treating SMA, Dr. Sumner says.
The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials of SMA therapies.
«We are now able to make biochemical measurements that will help us objectively assess functional changes in children and adults with SMA, and, hopefully, detect early signals of therapeutic success in new drug trials for this devastating disease.»
Seven - year - old Sophie with spinal muscular atrophy (SMA) is receiving the only medication specifically approved for SMA through her parent's work - sponsored drug plan.