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First-ever Oral Medicine Designed for Spinal Muscular Atrophy Patients gets FDA’s Approval

Despite the stress and stigma associated with the on-going Coronavirus pandemic, here is good news for spinal muscular atrophy patients. The U.S. Food and Drug Administration (FDA) has granted approval to the first-ever oral medication for the spinal muscular atrophy patients, giving them the hope to fight the disease. This medicine is called Evrysdi (risdiplam) which was first approved some two months ago to be used on patients.

It would be a part of the home care plan of spinal muscular atrophy patients. It could be given using a feeding tube or the mouth, directly.

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This approval was given on the basis of two clinical trial results, one of which was FIREFISH. This was open-label research having 21 participants all of which were aged 6.7 months at the time of study started.

Within one year, 41% of the patients were able to sit on their own for at least five seconds. After two years, 81% of participants didn’t need a permanent ventilation system. The U.S. Food and Drug Administration (FDA) has called it a highly significant improvement while recovering from such a disease.

The second study to get this approval was called “SUNFISH”. It was a randomized and placebo trial with 180 patients between the ages of 2 to 25. Within one year, the motor function of these patients was improved significantly compared to the placebo group.

The spinal muscular atrophy patients with infant onset of the symptoms might also experience lungs related infections. FDA is warning that people who are using the newly approved medicine Evrysdi should make sure not to use it along with other medicines that could trigger Evrysdi to boost plasma concentrations.

Spinal muscular atrophy (SMA) is a combination of different medical conditions that are associated with genetic linkage. These conditions typically cause weakness.

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The most common target of this condition are children but it can also affect the adults. It is a relatively rare medical condition which is why most people still don’t know about it.

Spinal muscular atrophy has four types. Based on the information about the development of this disease, a patient is categorized into any of the below-mentioned types.

  •  Type 1 (severe) SMA (Werdnig-Hoffmann Disease)
  • Type 2 (intermediate) SMA
  • Type 3 (mild) SMA (Kugelberg-Welander or Juvenile Spinal Muscular Atrophy)
  • Type 4 (adult) SMA

Although the spinal muscular atrophy patients don’t have a high-risk group in terms of race, ethnicity, or gender some people are more likely to get it. However, with this new medicine, there is a hope that these patients may get better someday.

There are minimum side effects expected from Evrysdi. Some of them are; diarrhea, rashes, fever, and ulcers. Some patients may experience urinary tract infections and arthralgia.



Areeba Hussain

The author is a fulltime medical and healthcare writer. She graduated in Medical Microbiology and Immunology with distinction. Her areas of prime interest are medicine, medical technology, disease awareness, and research analysis. Twitter @Areeba94789300

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