Spinal Muscular Atrophy Drug Pipeline - 2012

Jun 29, 2012

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Clinical Development. Preclinical: Discovery. Spinal Muscular Atrophy Drug Pipeline - 2012. FDA Approval. Basic Research Seed Ideas. Identification Optimization Safety & Manufacturing. Phase I Phase II Phase III. IND. Trophos/Olesxime. Repligen/Quinazoline*.

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neurological disorders
riluzole phenylbutyrate
paratek tetracycline
smn regulators
ninds benzimidazoles harvard
sma valproic acid

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Spinal Muscular Atrophy Drug Pipeline - 2012

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Clinical Development Preclinical: Discovery Spinal Muscular Atrophy Drug Pipeline - 2012 FDA Approval Basic Research Seed Ideas Identification Optimization Safety & Manufacturing Phase I Phase II Phase III IND Trophos/Olesxime Repligen/Quinazoline* ISIS - Biogen/Antisense* CSC/Motor Neuron* OSU/Gene Therapy* NINDS/Indoprofen Paratek/Tetracycline* *Projects with FSMA FundingInvolvement PTC - Roche/SMN Regulators Novartis/SMN Regulators Repurposed Drugs Studied for SMA Valproic Acid* Riluzole Phenylbutyrate* Hydroxyurea Salbutamol Scripps/SMN Regulators NCGC - IU/SMN Regulators NINDS/Benzimidazoles Harvard /SMN Regulators IU = Indiana University NCGC = NIH Chemical Genomics Center NINDS = National Institute of Neurological Disorders and Stroke

Spinal Muscular Atrophy

Spinal Muscular Atrophy. Occupational and Physical Therapy Jo Anne Maczulski, MA, OTR/L Kristin Krosschell, PT, MA, PCS. What is SMA?. Loss of function of spinal motor neurons causing progressive weakness of muscles Muscles include extremities, respiratory muscles Sensation is normal

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Pipeline Review on Spinal Muscular Atrophy Therapeutic Indus

“Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2014” report identifies emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.

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Spinal muscular atrophy

Spinal muscular atrophy. Type II& III: 1:24,000 Type II may sit but cannot walk Type III (Kugelberg-Welander disease) presents at 2-3 years of life Unrecognized nocturnal hypoxemia or hypoventilation [Bach and Wang 1995]

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Spinal Muscular Atrophy

By: Afif Hossain. Spinal Muscular Atrophy. A Detailed Look at the Mechanism of a Neurodegenerative Disease That Causes Muscle Deterioration. Introduction To SMA. Atrophy- the medical term for wasting or shrinkage, which is what generally happens to muscles when they’re not active

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Spinal Muscular Atrophy

Spinal Muscular Atrophy. Katerina Barker. Pathophysiology. Atrophy is the wasting away of muscles due to the lack of use. Not using your muscles make them weak. Causes and Symptoms. Causes could include; lack of use or exercise, mutations, poor nourishment, or poor circulation.

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Spinal Muscular Atrophy

Spinal Muscular Atrophy. By Robert Johnson . symptoms Infant. Can have a breathing difficulty Difficulty feeding, food may go down windpipe instead of stomach Floppy infant, poor muscle tone Lack of head control Little movements. Symptoms child. Much less symptoms then infant

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Spinal Muscular Atrophy SMN1

Spinal Muscular Atrophy SMN1. Billy Baader - Genetics 677. Gubitz et al. The SMN Complex. Experimental Cell Research(2004) 296: 51-56. Medline Plus (2009) Spinal Muscular Atrophy retrieved Feb 3, 2009 from: http://www.nlm.nih.gov/medlineplus/spinalmuscularatrophy.html. What is SMA?.

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New Compounds for Spinal Muscular Atrophy

New Compounds for Spinal Muscular Atrophy. CONFIDENTIAL. Jill Heemskerk, PhD Office of Translational Research National Institute of Neurological Disorders and Stroke. Why NIH Chose SMA for a Drug Development Pilot. Urgent unmet medical need Number one genetic killer of infants

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Spinal Muscular Atrophy CHRISTIAN SIMS

Spinal Muscular Atrophy CHRISTIAN SIMS. Symptoms. infants with SMA type I are born with very little muscle tone, weak muscles, and feeding and breathing problems. Often, weakness is first felt in the shoulder and leg muscles. Weakness gets worse over time and eventually becomes severe.

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Spinal muscular atrophy

Lise Caget Bodor Gergely. Spinal muscular atrophy. SMA. JPEMS 2014. Introduction. A heterogenous group of disorders . Various degrees of severity Collective incidence of 1/10,0 00 . Most common genetic cause of infant death. Signs&Symptoms. Areflexia.

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CHILDREN WITH SPINAL MUSCULAR ATROPHY

SCORD 2011. www.csma.org.ua. Formation of national understanding of rare diseases: from to. National Program. concept. Vitaliy MATYUSHENKO President of Foundation CSMA, Ukraine. CHILDREN WITH SPINAL MUSCULAR ATROPHY. SCORD 2011.

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Spinal Muscular Atrophy

Spinal Muscular Atrophy. By Robert Johnson. symptoms Infant. Can have a breathing difficulty Difficulty feeding, food may go down windpipe instead of stomach Floppy infant, poor muscle tone Lack of head control Little movements. Symptoms child. Much less symptoms then infant

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Spinal Muscular Atrophy

Spinal Muscular Atrophy. the number one genetic killer of infants and toddlers. Leena Shah Human Anatomy April 21, 2010. Samir died at 7 ½ months. Hailey Mae died at the age of 2. Owen died at 19 weeks and 6 days. Baylee died at 15 months. Spinal Muscular Atrophy (SMA).

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Spinal Muscular Atrophy Pipeline Review H2 2015

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Spinal Muscular Atrophy (SMA) Therapeutics Pipeline, Market Analysis and Forecast for the Drug Candidates in the Latest Stage of Development.

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Spinal Muscular Atrophy Market to Exhibit Steadfast Expansion During 2018-2026

The global spinal muscular atrophy market size was valued at US$ 884 million in 2017 and is expected to witness a robust CAGR of 12.4% over the forecast period (2018–2026).

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Spinal Muscular Atrophy Market Size

According to Renub Research forecast the global Spinal Muscular Atrophy Market to grow at a CAGR of 14% in the coming years. This high growth rate is primarily due to good reimbursement options in western countries, declining drug prices and projected launch of new drugs. One of the major growth drivers witnessing the growth of spinal muscular atrophy market is growing awareness of diagnosis and treatment of spinal muscular atrophy during the forecast period. For More Information: https://www.renub.com/global-spinal-muscular-atrophy-market-nd.php

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Spinal Muscular Atrophy Market to grow at a CAGR of 14%

Spinal muscular atrophy (SMA) is one of the most common genomic disorders which are caused due to the loss of specialized nerve cells known as motor neurons in the brainstem and spinal cord.

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What is spinal muscular atrophy and what is its tratment

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Spinal Muscular Atrophy Market

Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement.

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Spinal Muscular Atrophy Market Size & Share|Delveinsight

DelveInsight's Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast-2030 report delivers an in-depth understanding of the Spinal Muscular Atrophy, historical and forecasted epidemiology as well as the Spinal Muscular Atrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Spinal Muscular Atrophy (SMA) Treatment Market Research and Forecast 2019-2025

The global spinal muscular atrophy (SMA) treatment market is projected to exhibit considerable growth during the forecast period. The high prevalence of this disorder, a rapid rise in the R&D activities related to the development of therapies and treatment options by the major market players is a major factor anticipated to fuel the market during the forecast period.

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