Caring for a child with SMA is not only taxing on a family emotionally and physically, but it can also be quite taxing on your finances. Even if you have great health insurance (which let’s be honest, is rare), having a full-time caregiver can be incredibly expensive—whether that’s an outside support worker or a parent …
Potential Target for Severe Asthma, Other Chronic Inflammatory Diseases Identified
Researchers recently published new insights into the processing of the cystic fibrosis transmembrane conductance regulator protein and possible new therapeutic targets for cystic fibrosis.
(Blackboard shows foot with descriptions pointing to toes including 'Went to market', 'Stayed home', etc.
Pinner created this all-terrain walker for pinner's daughter, who has mild cerebral palsy. Great on sand and in shallow water.
Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children's National
New Post: Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children's National https://alsnewstoday.com/2018/03/02/retrophin-horizon-donate-3-million-to-childrens-national-rare-disease-institute?utm_content=buffere0a0f&utm_medium=social&utm_source=pinterest.com&utm_campaign=buffer
Spinal Muscular Atrophy (SMA) is a motor neuron disease, affecting the voluntary muscles? Check out this infographic for more SMA facts!
August is Spinal Muscular Atrophy Awareness Month. Useful facts about SMA
GD1 Children's Quality of Life Improved on Cerezyme Therapy, Study Says
New Post: Enzyme Replacement Therapy Improves Quality of Life in Children with GD1, Study Shows https://gaucherdiseasenews.com/2018/03/22/cerezyme-improves-quality-of-life-young-children-gaucher-disease-type-1-study
Adult With SMA Shares His Perspective on Spinraza
Ben Mattlin is in his and has had plenty of time to come to terms with having SMA. While he's excited about Spinraza, he doesn't want it for himself.
Horizon's FA Therapy Granted FDA Fast Track Designation Friedreich's Ataxia News
SMA and a Child at Play: Having Fun Ella's Way
Caregiver columnist Michael Casten shares the many unique ways his daughter Ella has learned to play, and the toys thought most useful for her development.
August is Spinal Muscular Atrophy Awareness Month
Spinal Muscular Atrophy Awareness Month (August Get Involved
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Why Motor Neurons in SMA so Sensitive to Protein's Loss Detailed in Possibly Key Study
New research details why a lack of the SMN protein in SMA patients quickly kills spinal motor neurons, a discovery that could help in treating the disease.
Mice Study Links Liver Genes to Low BMI in Endometriosis Patients
Researchers Identify a New Genetic Risk Factor for Multiple Sclerosis Development Multiple Sclerosis News Today
The PEComa clinical research study is for men and women over the age of 18 diagnosed with advanced malignant perivascular epithelioid cell cancer or PEComa.This study will evaluate the safety and potential benefits of using an investigational medication to treat your disease. Read more about our study here: http://www.mypecomastudy.com/the-pec-001-study/ For more information, visit our website to locate a participating site near you: http://www.mypecomastudy.com/current-study-locations/