Biogen and Ionis Pharmaceuticals recently provided an update on the clinical development of nusinersen (IONIS-SMNRx), which is currently being tested in Phase 3 clinical trials: Dear members of the SMA community, As we approach the upcoming Cure SMA conference in Anaheim, we want to provide an update on the status of the nusinersen program and
Biogen and Ionis Release Spring 2016 Community Update Read More »
The SMA drug pipeline has grown dramatically in just over a decade. Of the 18 programs in the pipeline, six of these are in clinical trials, and several of those are in Phase 2 and Phase 3 clinical trials. Reaching and achieving FDA approval is a major step in our path to a treatment and
2016 Update on SMA-FDA Interactions Read More »
The 2016 Annual SMA Conference Booklet has been posted. The booklet includes: A complete schedule with information about the special events we’ve got planned for each evening including: the Meet and Greet and Family Fun Fest, the Family Friendly Research Poster Session, and Saturday Night at Disneyland Park! Information on Saturday afternoon’s panels focusing on
2016 Annual SMA Conference Booklet Now Posted Read More »
Novartis recently provided the following update on clinical trials for LMI070, an orally available SMA drug that corrects SMN2 splicing: “We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial,
Update on the Clinical Development of LMI070 Read More »
The Spring 2016 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced eight grants totaling $890,000 for basic research, and two grants totaling $100,000 for clinical care research. This issue introduces our ten grantees and gives background on each of their projects: what the project is,
Spring 2016 Issue of Compass Now Available Read More »
Cure SMA has awarded a $300,000 drug discovery grant to Charlotte Sumner, MD, at Johns Hopkins University, in collaboration with Imago Pharmaceuticals. The award is for the project, “Preclinical Development of a JNK Drug Candidate to Alter Disease Progression in SMA.” The Importance of Combination Therapies As the SMA drug pipeline has grown in breadth,
Just added to this year’s conference schedule: Saturday Night at Disneyland Park! We’re very excited to announce that conference attendees will get to take part in this special evening of Disney fun, supported by Biogen. All registered participants will receive complimentary Saturday Disney Entrance Tickets to Disneyland Park. The ticket also incudes a special designated
Announcing an Evening of Fun at Disneyland Park Read More »
Cure SMA has released the full schedule for the 2016 Annual SMA Conference, which will be held in Anaheim, CA at Disneyland, June 16-19. The schedule includes the detailed timeline of workshops and events, including the Meet & Greet and Family Fun Fest, the Family Friendly Research Poster Session, and the PJ Party & Movie
Cure SMA Releases Full Schedule for the 2016 Annual SMA Conference Read More »
Cure SMA has awarded a $70,000 research grant to Dr. Christine DiDonato at Lurie Children’s Hospital of Chicago for her project, “Assessing mediators of muscle weakness in SMA mice.” Individuals with SMA do not produce survival motor neuron (SMN) protein at high enough levels, leading to debilitating and often fatal muscle weakness. One of the
Today, AveXis, Inc. presented an interim analysis of data as of April 1, 2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, presented the data at the
