Cure SMA will host the world’s largest regular gathering on spinal muscular atrophy next month in Dallas, bringing together leading SMA researchers and clinicians, as well as families living with the disease.
The 2018 Annual Cure SMA Conference is set for June 14-17 at the Hilton Anatole Hotel.
Its first three days also coincide with the 22nd Annual SMA Researcher Meeting, whose agenda features a special session onJune 14 moderated by Richard Finkel, MD, chief of the neurology division at Nemours Children’s Hospital in Orlando.
During the session, “Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity,” seven SMA experts will discuss new challenges and opportunities, focusing on prioritizing research endeavors to address unmet medical needs for SMA patients. Speakers include Ohio State University’s Arthur Burghes, PhD; and Stephen Kolb, MD, PhD; Harvard Medical School’s Basil Darras, MD; and John Hopkins School of Medicine’s Thomas Crawford, MD.
Other scientific sessions on the agenda include “Modifiers of Disease and Therapeutic Targets,” led by Elliot Androphy, MD; “SMN Function and Expression,” moderated by Adrian Krainer, PhD; “SMA Pathology and Tissue Requirements,” led by Rashmi Kothary, PhD; and “Clinical Research Studies for SMA,” moderated by Crawford.
Biogen, producer of the approved SMA therapy Spinraza (nusinersen), is sponsoring a June 14 lunch symposium, while Genentech/Roche plans a June 15 breakfast symposium. That’ll be followed by a June 16 breakfast symposium sponsored by AveXis, which last month was acquired by Swiss pharma Novartis for $8.7 billion. Genentech and Roche are currently testing in several clinical trials RG7916, an oral SMN2 splicing modifier; AveXis is advancing AVXS-101, a potential SMA gene therapy, in its clinical studies.
At this year’s Family Friendly Researcher Poster Session, 27 posters will be presented, according to the Cure SMA website. Their subjects range from whether fat intake makes a difference for SMA patients to the importance of newborn screening for the neuromuscular disease.
Earlier this month, Indiana became the fourth state to officially adopt newborn screening for SMA; Missouri, Minnesota and Utah have already done so.
SMA affects roughly 1 in 11,000 babies. Likewise, about 1 in 50 Americans — or around 8 million people — are genetic carriers for SMA. But because it’s an autosomal recessive disease, both parents must usually be carriers for a child to inherit the condition.
“Indiana is at the leading edge of an effort to save the lives of babies born with spinal muscular atrophy,” Cure SMA President Kenneth Hobby said in a press release. “SMA is currently the number-one genetic cause of death for infants under two years of age, but we now have an historic opportunity to change that through newborn screening and early treatment.”
With an annual budget of $11 million, Cure SMA has spent about $70 million to date on research, Hobby said during a recent speech at the World Orphan Drug Congress in Maryland.
This year’s agenda in Dallas features 42 family workshops covering a variety of topics. These include “Yoga Therapy for Teens & Adults with SMA,” “Care for the Caregiver,” “Healing the Grieving Heart” and “Breathing Strong All Day Long: BiPAPs and Ventilators.”
But the conference is not all about science. For kids and teenagers, there’s also a dance party on June 14, and a pajama party and movie night on June 15.
More than 2,000 people, including 450 researchers from nearly 100 organizations, attended last year’s Cure SMA conference in Orlando. That event featured 52 family workshops and 130 research presentations; it was supported by 156 sponsors and 23 exhibitors.