News : Spinal Muscular Atrophy

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Lincolnshire Charity Plea for Auction Donations

5/23/2013 9:08 AM EST  -   Shooting Star PR
News release

21st May 2013
The Ally Cadence Trust, based in Lincolnshire, is asking businesses to donate items for auction at its biggest annual fundraising event.

The Butterfly Ball, which is being held at the Petwood Hotel in Woodhall Spa on 28th September, will be hosted by Emma and Karl Humphries, the founders of the Ally Cadence...

Families of SMA and Nationwide Children’s Announce Multi-Million Dollar Award fr...

3/13/2013 1:00 PM EST  -   Families of SMA
Elk Grove Village, IL. March 13, 2013--Nationwide Children’s Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA).

This three-year multi-million dollar cooperative...
Mum Emma Humphries holds baby Ally Cadence during one of her hospital stays
 

Shooting Star to ACT for Local Charity

1/30/2013 11:15 AM EST  -   Shooting Star PR
News release

30th January 2013
A charity started in the name of a little girl who died from a rare muscular condition will be given free publicity after being chosen as Charity of the Year by Shooting Star PR.

Each year the Lincoln-based public relations agency provides free advice and expertise to a different local charity or non-profit organisation which would not otherwise be able to afford ongoing...

Pfizer Licenses Families of Spinal Muscular Atrophy Quinazoline Drug Program fro...

1/3/2013 9:15 AM EST  -   Families of SMA
Elk Grove Village, IL. January 3, 2013.

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).

Families of SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This was the first drug...

Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Resear...

12/31/2012 10:00 AM EST  -   Families of SMA
Elk Grove Village, IL. December 31, 2012--Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, which includes: Basic Research to reveal the best ways of making SMA drugs; Drug Discovery to make practical new drugs, and; Clinical Trial resources to help test new drugs.

Families of SMA committed new funding to the...

Families of Spinal Muscular Atrophy Awards $710,000 for Basic Research to Identi...

12/28/2012 10:00 AM EST  -   Families of SMA
Elk Grove Village, IL. December 28, 2012--Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program.  Continued investment in basic research leads to greater understanding of the exact nature, causes, and consequences of SMA.  This knowledge is key to ensuring that the most effective SMA treatments can be identified and...

Repligen Initiates Phase 1b Trial of RG3039 for Spinal Muscular Atrophy Licensed...

9/26/2012 4:08 PM EST  -   Families of SMA
Elk Grove Village, IL. September 26, 2012.

Repligen Corporation has initiated a Phase 1b clinical study of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).  The primary objectives of the study are to further evaluate the safety and plasma pharmacokinetics (PK) of multiple doses of RG3039 in healthy volunteers.

Repligen licensed RG3039 in 2009 from Families...

Families of SMA Announces $1.4 Million Planned for Next Round of Spinal Muscular...

6/7/2012 3:00 PM EST  -   Families of SMA
Elk Grove Village, IL. June 7, 2012.

Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program. 

The organization is planning to award $1.4 Million in new research funding over the next 6 months.  This new round of research funding will be allocated into three areas: 1)Basic Research to...

Families of SMA Announces $700,000 Funding for Novel Drug Program at Newly Creat...

5/10/2012 3:30 PM EST  -   Families of SMA
Elk Grove Village, IL. May 10, 2012.

This new program will focus on optimizing small molecule drug candidates which increase the SMA back-up gene in order to make them ready for human clinical trials. It is well demonstrated that increasing production from the back-up gene can lead to improvement in mouse models of SMA. Small molecule drug candidates have particular advantages: mainly ease of use by oral dosing and...

Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for S...

5/2/2012 4:00 PM EST  -   Families of SMA
Elk Grove Village, IL. May 2, 2012-Families of SMA is pleased to announce the award of up to $750,000 for an important new grant to Dr. Brian Kaspar at Nationwide Children’s Hospital. This award will support preclinical development of a CNS-delivered Gene Therapy for Spinal Muscular Atrophy. With funding from FSMA, Dr. Kaspar’s team will initiate the studies needed for an Investigational New Drug (IND) application...