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
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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
The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them. During the event, families
Cure SMA Announces 2016 Family Friendly Poster Session Presenters Read More »
Recently, Cure SMA confirmed three new board members and several new committee members. These new members will provide additional leadership in the areas of advocacy, FDA/regulation, and research. We extend a warm welcome to new board members Annie Kennedy, Spencer Perlman, and John Porter, PhD. We also extend a warm welcome to new committee members
Cure SMA Welcomes New Board and Committee Members Read More »
Ionis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with spinal muscular atrophy (SMA) at the American Academy of Neurology (AAN) meeting. The data reported show that there have been no new events, as defined by progression to permanent ventilation or death, in the study since December
Ionis Releases Data Update from Phase 2 Study in Infants with SMA Read More »
