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SpinalMuscularAtrophy news

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy


News-Medical.net - Jan 10, 2017
Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy. They report they have evidence that an experimental medicine aimed at this target works ...
 

Nusinersen Found Promising for Spinal Muscular Atrophy


LWW Journals - Jan 6, 2017
Administration of multiple intrathecal doses of nusinersen showed acceptable safety and tolerability, pharmacology consistent with its intended mechanism of action, and encouraging clinical efficacy for spinal muscular atrophy, researchers reported in ...
 

FDA approves Biogen's spinal disease therapy


The Boston Globe - Dec 24, 2016
“There has been a longstanding need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life,” said Dr. Billy Dunn, director of the neurology products ...
 

Spinal Muscular Atrophy - premium items on eBay

Spinal Muscular Atrophy: Disease Mechanisms and Therapy by Charlotte J Sumner


 

NEW X-Linked Spinal Muscular Atrophy: The Linkage, The Gene & SMN Complex


 

NEW X-Linked Spinal Muscular Atrophy: The Linkage, the Gene & Smn Complex by Yar


 

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Spinal Muscular Atrophy books

Laughing at My Nightmare


by: Shane Burcaw

With acerbic wit and a hilarious voice, Shane Burcaw's Laughing at My Nightmare describes the challenges he faces as a twenty-one-year-old with spinal muscular atrophy. From awkward handshakes to having a girlfriend and everything in between, Shane handles his situation with humor and a "you-only-live-once" perspective on life. While he does talk about everyday issues that are relatable to teens, he also offers an eye-opening perspective on what it is like to have a life threatening disease.
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Spinal Muscular Atrophy: Disease Mechanisms and Therapy


Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development.

This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development.
>> More info on Amazon

The Me in the Mirror


by: Connie Panzarino
Writer, activist and artist Connie Panzarino was born in 1947 with the rare disease Spinal Muscular Atrophy Type III, formerly called Amytonia Congenita. Throughout a childhood filled with both pain and joy, she strove to define herself: "I knew I was different. Now I had a name for the difference, like being Italian or Jewish. I was an Amytonia. I didn't understand if that meant that I would never walk, or if all it meant was lack of muscle tone. I didn't know that most children with this disease die before they're five years old.
>> More info on Amazon

Miracle Boy Grows Up: How the Disability Rights Revolution Saved My Sanity


by: Ben Mattlin
"An insightful, poignant, light-hearted and often hilarious memoir" (Jay McInerney).  Ben Mattlin lives a normal, independent life. Why is that interesting? Because Mattlin was born with spinal muscular atrophy, a congenital muscle weakness from which he was expected to die in childhood. Not only did Mattlin survive, he became one of the first students in a wheelchair to attend Harvard, from which he graduated and became a professional writer. His advantage? Mattlin's life happened to parallel the growth of the disability rights movement, so that in many ways he did not feel he was disadvantaged at all, merely different.
>> More info on Amazon

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Spinal Muscular Atrophy


This video was created with the intent to research SMA.-- Created using PowToon -- Free sign up at http://www.powtoon.com/join -- Create animated videos and ...
 

Roche in Spinal Muscular Atrophy


Spinal Muscular Atrophy (SMA) is the leading genetic cause of death in infants and toddlers and is associated with a high rate of childhood mortality in its most ...
 

Finding a cure for spinal muscular atrophy (SMA)


Stephen Clark is on a mission to help his granddaughter Scarlet, who suffers from a rare, genetic condition call spinal muscular atrophy, or SMA. ◂ WXYZ 7 ...
 

Spinal Muscular Atrophy Treatment at Nationwide Children's -- Brett & Paige


Help us help kids like Brett. Please give by visiting ...
 

Reagan Goforth, Spinal Muscular Atrophy | Stem Cell Treatment Testimonial


Reagan was clinically diagnosed with SMA at 6 months after extensive genetic testing due to delayed motor function and and overall delay in hitting infant ...