Oral Therapy Has Changed The Life of This Little Girl Living with Spinal Muscular Atrophy

Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is a rare disease that causes progressive muscle loss and resultant muscle weakness. It is caused by a lack of motor neurons. It…

Continue Reading Oral Therapy Has Changed The Life of This Little Girl Living with Spinal Muscular Atrophy
Spinraza Linked to Immune Cell Abnormalities in Two Spinal Muscular Atrophy Patients
source: pixabay.com

Spinraza Linked to Immune Cell Abnormalities in Two Spinal Muscular Atrophy Patients

According to a story from SMA News Today, a recent case study has reported instances in two spinal muscular atrophy (SMA) patients in which repeated administrations of the therapy nusinersen…

Continue Reading Spinraza Linked to Immune Cell Abnormalities in Two Spinal Muscular Atrophy Patients
Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients
source: pixabay.com

Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients

by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…

Continue Reading Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients
Gene Therapy in Mouse Model Gives Insight into Possible SMA Treatments
source: pixabay.com

Gene Therapy in Mouse Model Gives Insight into Possible SMA Treatments

by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…

Continue Reading Gene Therapy in Mouse Model Gives Insight into Possible SMA Treatments
This Organization is Offering Spinal Muscular Atrophy Patients Financial Aid for Treatment
source: pixabay.com

This Organization is Offering Spinal Muscular Atrophy Patients Financial Aid for Treatment

According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…

Continue Reading This Organization is Offering Spinal Muscular Atrophy Patients Financial Aid for Treatment
Medication Spinraza Shows Promising Benefits in Pre-Symptomatic SMA Patients
source: pixabay.com

Medication Spinraza Shows Promising Benefits in Pre-Symptomatic SMA Patients

By Lauren Taylor from In The Cloud Copy Biogen announced new data from the NURTURE trial of pre-symptomatic patients with spinal muscular atrophy (SMA). SMA is a rare, autosomal recessive…

Continue Reading Medication Spinraza Shows Promising Benefits in Pre-Symptomatic SMA Patients
Scientists Can Identify Schwann Cells Using Novel Bar Code Technique
Image by Markus Spiske from Pixabay

Scientists Can Identify Schwann Cells Using Novel Bar Code Technique

Scientists and researchers have long known how important glial cells are to the nervous system. Not only do these cells make up a majority of cells within the central nervous…

Continue Reading Scientists Can Identify Schwann Cells Using Novel Bar Code Technique

European Commission Grants Conditional Approval for Spinal Muscular Atrophy Treatment

Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…

Continue Reading European Commission Grants Conditional Approval for Spinal Muscular Atrophy Treatment
RNA Therapy Holds Promise for Treating SMA and Other Rare Diseases
source: pixabay.com

RNA Therapy Holds Promise for Treating SMA and Other Rare Diseases

by Jodee Redmond from In the Cloud Copy   Emma Larson’s parents were not particularly concerned when their daughter wasn’t standing or walking by her first birthday. They figured that plenty of children had not reached…

Continue Reading RNA Therapy Holds Promise for Treating SMA and Other Rare Diseases
Man Runs 400 Laps Around His Own House to Help Spinal Muscular Atrophy Patient
source: pixabay.com

Man Runs 400 Laps Around His Own House to Help Spinal Muscular Atrophy Patient

According to a story from The Charlotte Weekly, local resident Kevin Tobin recently ran 400 laps around his house---the equivalent of a marathon---to help raise money for Dan Donoher, a…

Continue Reading Man Runs 400 Laps Around His Own House to Help Spinal Muscular Atrophy Patient