Educational Resources

While we are dedicated to educating the public about SMA and its effects, we also want to provide healthcare professionals with the necessary tools to enhance their knowledge of the disease. It is our hope that through shared data and information, our research community will enrich the quality of care and support available to SMA families.

Educational Opportunities 

Medical Advisory Council members are available to provide continuing medical education to medical providers. Please contact Page Kirkpatrick at page@curesma.org for further information.

Cure SMA Conference Medical Presentations

SMA Clinical Care Evidence

Cure SMA-Funded Articles

  1. Respiratory muscle function in infants with spinal muscular atrophy type I. Finkel RS, Weiner DJ, Mayer OH, McDonough JM, Panitch HB. Pediatr Pulmonol. 2014 Apr 29. doi: 10.1002/ppul.22997.
  2. Assessing the Needs of the SMA Population: Survey Results of Health Care Providers and Families. Halanski MA, Patterson KG, Sund SA, Makholm LM, Schroth MK. SAGE Open. October-December 2014: 1–5. 
  3. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands. Finkel R, Bertini E, Muntoni F, Mercuri E; ENMC SMA Workshop Study Group. Neuromuscul Disord. 2015 Apr 28. pii: S0960-8966(15)00134-0.
  4. Responses to Fasting and Glucose Loading in a Cohort of Well Children with Spinal Muscular Atrophy Type II. Davis RH, Miller EA, Zhang RZ, Swoboda KJ. J Pediatr. 2015 Oct 7. pii: S0022-3476(15)01027-6. 
  5. Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study. Qian Y, McGraw S, Henne J, Jarecki J, Hobby K, Yeh WS. BMC Neurol. 2015 Oct 24;15(1):217.  
  6. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Iannaccone ST, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Cudkowicz ME, McGovern MM, McNeil DE, Finkel R, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes AH, Bartlett A, Kissel JT; NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. Ann Clin Transl Neurol. 2016 Jan 21;3(2):132-45. doi: 10.1002/acn3.283. eCollection 2016 Feb. 
  7. Nutritional Status and Nutrient Intake Challenges in Children With Spinal Muscular Atrophy. Mehta NM, Newman H, Tarrant S, Graham RJ. Pediatr Neurol. 2016 Apr;57:80-3. doi: 10.1016/j.pediatrneurol.2015.12.015. Epub 2015 Dec 24. PMID: 26803333 
  8. What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients. Hunter M, Heatwole C, Luebbe E, Johnson NE. J Neuromuscul Dis. 2016 Aug 30;3(3):425-429. 
  9. Opening the window: The case for carrier and perinatal screening for spinal muscular atrophy. Burns JK, Kothary R, Parks RJ. Neuromuscul Disord. 2016 Sep;26(9):551-9. 
  10. An Evaluation of a Continuing Education Program for Family Caregivers of Ventilator-Dependent Children with Spinal Muscular Atrophy (SMA). Boroughs D. MDIP. Children 201, 3, 33; doi:10.3390/children4050033. 

Other Clinical Care Evidence

The Lancet Published Data from Phase 2, open-label trial of Spinraza in Infants 

'Dawn of a New Therapeutic Era for Spinal Muscular Atrophy' - The Lancet 

Correlation Between the Degree of Correction of Neuromuscular Scoliosis and Patient Quality of Life 

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool

ACOG Committee Opinion - Carrier Screening for Genetic Conditions

Medical Education Tools

On September 30, 2016, experts in SMA care met to present and discuss the latest discoveries and options for optimizing care. The meeting was organized by The University of Pennsylvania's Penn Medicine and was co-sponsored by AveXis, Inc. and the Orphan Disease Center. 

The following information is from the 2016 Spinal Muscular Atrophy (SMA) Symposium on Optimizing Care. Cure SMA has permission from the conference and presenters to share the presentations and audio files below. 

Neurology

SMA Diagnosis/Pathology - John Brandsema, MD, Div. of Neurology, The Children's Hospital of Philadelphia 

Novel Disease Modifying Therapeutics - Basil Darras, MD, Div. of Neurology, Boston Children's Hospital

SMA Functional Assessment - Allan Glanzman, PT, DPT, PCS, Dept. of Physical Therapy, The Children's Hospital of Philadelphia

Person-Reported Outcome Measures - Amy Pasternak, PT, DPT, PCS, Dept. of Physical Therapy, Boston Children's Hospital

Decision Making in SMA - Thomas Crawford, MD, Div. of Neurology, Johns Hopkins Children's Center

Pulmonology

Airway Clearance - When to Start, What to Use - Richard Shell, MD, Div. of Neurology, Nationwide Children's Hospital 

Chronic Respiratory Failure & When to Start Ventilation - Howard Pantich, MD, Div. of Pulmonogly, The Children's Hospital of Philadelphia

Acute Management - Home and Inpatient - Robert Graham, MD, Div. of Critical Care, Boston's Children Hospital 
Respiratory Range of Motion - Hank Mayer, MD, Div. of Pulmonogly, The Children's Hospital of Philadelphia 
The Role of Exercise in SMA - Jacqueline Montes, PT, EdD, NCS, Dept. of Neurology, Columbia University Medical Center 

Calorie Supplementation - When to Initiate & How - Donna DiVito, RD, CNSD, Div. of Gastroenterology and Nutrition, The Children's Hospital of Philadelphia 

Gastrostomy Tube Management/Fundoplication - Thane Blinman, MD, Div. of General Surgery, The Children's Hospital of Philadelphia 

Ortho

Management of Contractures of the Upper Limb in SMA Patients - Daniel Zlotolow, MD, Div. of Orthopedics, Shriners Hospital for Children

Options for Management - Lower Extremity - David Spiegel, MD, Div. of Orthopedics, The Children's Hospital of Philadelphia
Novel Approaches for Assessing the Chest Wall & Spine - Robert Campbell, MD, Div. of Orthopedics, The Children's Hospital of Philadelphia
Options for Management - Growing Rods - Paul Sponseller, MD, Div. of Orthopedics, Johns Hopkins Children's Center
Options for Management - VEPTR - Patrick Cahill, MD, Div. of Orthopedics, The Children's Hospital of Philadelphia




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