Spinal Muscular Atrophy

Repligen Reports Positive Results From Phase 1 Clinical Trial for Spinal Muscular Atrophy (SMA)

Wed, 25 Apr 2012 07:36:43 -0700 ELK GROVE VILLAGE, Ill. — Repligen Corporation today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate … http://finance.yahoo.com/news/repligen-reports-positive-results-phase-143643559.html

Repligen Reports Positive Results from Phase 1 Clinical Trial of RG3039 for Spinal Muscular Atrophy

Wed, 25 Apr 2012 04:00:00 -0700 Repligen Corporation today announced positive results from a Phase 1 study to evaluate the pharmacokinetic and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy . http://finance.yahoo.com/news/repligen-reports-positive-results-phase-110000139.html

Repligen Announces Favorable Results from Phase 1 Clinical Trial of Experimental Treatment for Spinal Muscular Atrophy

Wed, 25 Apr 2012 00:56:07 -0700 TUCSON, Ariz., April 25, 2012 /PRNewswire-USNewswire/ — Repligen Corp., in Waltham, Mass., announced today that its experimental drug RG3039, designed to treat spinal muscular atrophy (SMA), was safe and well-tolerated in a phase 1 clinical trial. In addition, data suggested that the drug reached and acted on its biological target, an enzyme called DcpS. http://news.yahoo.com/repligen-announces-favorable-results-phase-1-clinical-trial-075607492.html

Repligen Reports Positive Results from Phase 1 Clinical Trial of …

Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA), a patient advocacy organization that funded and directed the preclinical development of RG3039 with an investment of more than $13 … http://www.spinalmuscularatrophy.info/2012/04/repligen-reports-positive-results-from.html

Spinal Muscular Atrophy Discovery

Spinal muscular atrophy (SMA) is a devastating hereditary disease marked by muscle weakness and lack of movement in children and infants. Low levels of a protein in nerve cells known as survival motor neuron protein, … http://biology.about.com/b/2012/04/13/spinal-muscular-atrophy-discovery.htm

Childhood spinal muscular atrophy: controversies and challenges …

Childhood spinal muscular atrophy: controversies and challenges. By – Prof Eugenio Mercuri MD, Enrico Bertini MD, Prof Susan T Iannaccone MD. http://www.thelancet.com/journals/laneur/article/PIIS1474-4422(12)70061-3/abstract

1/4″ Charity Rope Halter Black Spinal Muscular Atrophy

28 Apr 2012 03:35:05 GMT-07:00 $16.99
End Date: Tuesday May-1-2012 11:41:03 PDT
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Living With Spinal Muscular Atrophy: The True Story of Kassidy Jade Sears by…

28 Apr 2012 03:35:05 GMT-07:00 $8.13
End Date: Thursday May-24-2012 5:08:06 PDT
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NEW Living with Spinal Muscular Atrophy: The True Story

28 Apr 2012 03:35:05 GMT-07:00 $9.20
End Date: Monday May-21-2012 17:23:43 PDT
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Current Concepts in Childhood Spinal Muscular Atrophy, L. Merlini, C. Granata, E

28 Apr 2012 03:35:05 GMT-07:00 $91.62
End Date: Friday May-11-2012 22:33:05 PDT
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Living with Spinal Muscular Atrophy: The True Story of

28 Apr 2012 03:35:05 GMT-07:00 $15.01
End Date: Wednesday May-16-2012 16:28:56 PDT
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4 Responses to Spinal Muscular Atrophy

  • Anonymous says:

    How To Help/what Can I Do For My Former Teacher? When i was in 4th grade, i had this awesome teacher named mr. Casten. He was by far the best techer i’ve ever had. He had two beautiful children. After that school year, he and his wife had another daughter named Ella. A couple years later, when my younger brother got mr. Casten for his 4th grade teacher, something went wrong with Ella. She was diagnosed with SMA, or Spinal Muscular Atrophy. It is a rare and deadly disease. With no cure, Ella will live a short life. Mr. Casten’s world was turned upside down. There was many fundraisers. Keep in mind, me and mr. Casten had a special, i guess, “connection” when he taught me. Today, the largest of all fundraisers was held. Many,many people showed up, even Keenan Cahill!! So, i just feel so bad for him, and it just breaks my heart. I’ve been wanting to help, but i dont know how. At the fundraiser, me and my best friend went and gave him a hug, and gave him Oreos (his favorite food). He said thank you for coming. I just really want to show my appreciation for him. And we had a conversation on facebook, here it is. Oh, by the way, he hates pears. And i love joking around with him. Me:Hi i know you dont want to facebook with me, but say hi to ella for me!! And eat some pears!! Bye!
    Him:I will say hi…I am holding her hand while she falls asleep…I will not, however, eat pears.
    Me:Hahahaha thanks 🙂
    It just touched my heart…he said he was holding her hand while she falls asleep…she’s now in the hospital…
    Oh, I’m tearing up. Thank you for all your help. Also, I’m only in 6th grade, so i can’t donate a ton of money or anything like that. THANK YOU!! ;(
    i think she’s 1 1/2 yrs

    • Curator says:

      The only thing you can do is be there for him. A little girl (nine) died of this and it came as a shock to everyone because she was doing so well! She was born with it and because of it hadn’t been able to walk or stand on her own but about a month before she pasted away for the first time ever she stood up on her own.

  • Why Is That People Complain About Black History Month But Dont Complain About The Other Months? So Black Americans cant have their own month? Why the double standards and hypocrisy? Heres a list of all other months. I would think that this list would keep everybody happy but I guess everyone is happy when Black History is not recognized. smh

    National Mentoring Month
    Thryroid Disease Awareness Month
    National Birth Defects Prevention Month (New York)
    National Glaucoma Awareness Month (Illinois)
    National Stalking Awareness Month [1]
    Geek Appreciation Month [2]

    [edit] February
    Black History Month (USA)
    National Eating Disorders Screening Program
    National Wise Mental Health Consumer Month
    National American Heart Month
    National Women’s Heart Health Day
    National Have-A-Heart Day: Feb. 14
    American Heart Association (Texas)
    Low Vision Awareness Month: Prevent Blindness America (Illinois)
    National Cardiac Rehabilitation Week (Wisconsin)
    National Burn Awareness Week (Ohio)

    [edit] March
    National Kidney Month
    National Nutrition Month[1]
    Women’s History Month
    Irish-American Heritage Month
    Greek-American Heritage Month
    National Brain Injury Awareness Month [2]
    Endometriosis Awareness Month
    Juvenile Arthritis Awareness Month
    Self Harm Awareness Month

    [edit] April
    Autism Awareness Month
    Jazz Appreciation Month
    National Poetry Month
    Mathematics Awareness Month[3]
    Sexual Assault Awareness Month
    National Volunteer Month
    Arab-American Heritage Month
    Tartan (Scottish-American) Heritage Month
    Donate Life Month- Organ and Tissue Donation

    [edit] May
    Rowena’s Eat Dessert First Month (http://www.rowenas.com/news)
    Brain Tumor Awareness Month (http://www.MilesForHope.org)
    Cystic fibrosis Awareness Month [4]
    South Asian Heritage Month
    Haitian Heritage Month
    Asian Pacific American Heritage Month
    Borderline Personality Disorder Month[citation needed]
    Asthma Awareness Month[5]
    National Pet Month
    Mental Wellness Month
    Science Month
    Celiac Awareness Month
    National Guide Dog Month (2008, 2009)
    Jewish American Heritage Month
    National Law Enforcement Appreciation Month
    National Masturbation Month[6]
    Social Media Month[7]
    Lupus Awareness Month (US)[8]

    [edit] June
    Caribbean American Heritage Month[citation needed]
    Gay/Lesbian Pride Month
    National Father’s Day Month

    [edit] July
    National Vehicle Theft Protection Month[citation needed]
    National Tickling Month
    French-American Heritage Month
    National wikipedia day

    [edit] August

    Spinal Muscular Atrophy awareness month

    [edit] September
    Arthritis Awareness Month (Canada)[3]
    Childhood Cancer Awareness month
    National Guide Dog Month (2010 to present)
    National Hispanic Heritage Month (September 15 to October 15)
    National Bourbon Heritage Month
    National Yoga Month
    National Preparedness Month[9]
    German-American Heritage Month (September 15-October 15)
    Polycystic Ovarian Syndrome Awareness Month

    [edit] October
    Rett Syndrome Awareness Month
    National Pork Month (United States) [10]
    Black History Month (UK)
    National Book Month[11]
    National Breast Cancer Awareness Month
    National CyberSecurity Awareness Month[12]
    Filipino American History Month
    LGBT History Month
    Nonprofit Career Month
    National Bullying Prevention Month
    National Fire Prevention Month
    National Domestic Violence Awareness Month
    Italian-American Heritage Month
    Vegetarian Awareness Month
    National Pregnancy and Infant Loss Awareness Month
    Selective Mutism Awareness Month
    National Pizza Month
    Lupus Awareness Month (Canada)[13]
    National Dental Hygiene Month[14]

    [edit] November
    Alzheimer Awareness Month
    National American Indian & Alaska Native Heritage Month[15]
    COPD Awareness Month
    Arts + Health Month
    National Adoption Awareness Month
    Nonprofit Awareness Month
    Epilepsy Awareness Month
    Pancreatic Cancer Awareness Month]
    Prematurity Awareness Month

    [edit] December

    National Bleeding Money Month

    • Curator says:

      I think it is retarded to have a black history month. Most of those other things you listed are to raise awareness for things like disorders and illnesses.

      The jews were treated worse then the blacks more recently and they don’t have a holocaust month. Atheists and every religion other than catholics were tortured in the Spanish Inquisition. Do they have a month? White people get the most disadvantages nowadays and we don’t have a month.

      If you don’t believe me go look at the requirements for standardized testing, if you are white you have to score higher on your exam in order to pass. High schools are allowed to have a Minority Student Union, Ebony Club, Black Honor Society. White clubs are not allowed due to racism. White people have a harder time getting on government assistance. I have tourettes and Autism and I am unable to get on government assistance. My disability lawyer even said (in joking) “too bad you are not black, then it would be easier to get you ssi”. There is a channel called BET and a magizine called Ebony. Do you see a white magazine or a white only tv channel? They even have their own award shows that only give awards to black actors, singers, etc. They are allowed to call themselves the “N” word without getting in trouble.

      Now I expect to get thumbs down. But just ponder this. If my yahoo avater was black would you have thumbed me down for saying these things? probably not.

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