Abstract

SPINAL MUSCULAR ATROPHY: OPTIMIZING CLINICAL OUTCOMES WITH NOVEL THERAPIES AND ROLE OF PHARMACISTS: A REVIEW

Payal Dasgupta*, Bhupen Kalita and Kamallochan Barman

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Abstract

Spinal muscular atrophy (SMA) is the most common fatal inherited disease in infants, with different types of SMA ranging from mild to extremely severe and terminal. For the most SMA types which typically affect since birth but in some cases don’t showcase the symptoms till adulthood. The progression may vary with the time of rapid progression and stasis, although the complete mental, physical and social well-being of the disease has been merely understood. The recent approval of novel therapies for the treatment of SMA has transformed the treatment paradigm and has the potential to increase survival in patients with SMA. Early diagnosis and initiation of therapy is imperative, and pharmacists can facilitate incorporation of these agents appropriately into the treatment plan for patients with SMA. Pharmacists must understand the safety and efficacy data, as well as real-world experience associated with early initiation of treatment, to provide patients, caregivers, and other health care providers on the multidisciplinary team with information about the benefits versus risks associated with available therapies. This article will also incorporate information, allowing readers to follow cases of patients with SMA and providing an opportunity to immediately implement the knowledge gained from the article.

Keywords: Spinal muscular atrophy, Neuromuscular disorder, Motor neurons, Diagnosis, Treatment, Pharmacist.