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Spinal muscular atrophy (SMA) is a hereditary neuromuscular disease that typically begins in infancy or childhood but can manifest at any age.
It is characterized by the irreversible and progressive degeneration of motor neurons in the spinal cord and brainstem. This results in a wide range of symptoms, in addition to which there is substantial variation in the rate of progression and disease prognosis.
Although early diagnosis and timely therapy can slow or prevent disease progression, disease-modifying therapies since 2016 have significantly advanced the management of SMA.
In a clinically focused program, Dr Perry Shieh, a neuromuscular neurologist from the University of California, Los Angeles, discusses the three medications currently approved by the US Food and Drug Administration: nusinersen, risdiplam, and onasemnogene abeparvovec.
He weighs the clinical benefits and key considerations for the use of each drug and emphasizes the need for shared decision-making with the patient.
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Professor, Departments of Neurology and Pediatrics, University of California, Los Angeles; Neuromuscular Neurologist, Ronald Reagan UCLA Medical Center, Los Angeles, California
Perry Shieh, MD, PhD, has disclosed the following relevant financial relationships:
Serve(d) as a speaker or a member of a speakers bureau for: Grifolis; Biogen; Genentech; CSL Behring; Alexion; Argenx; Catalyst
Received income in an amount equal to or greater than $250 from: Sarepta; Novartis; Biogen; Genentech; Alexion; Argenx; Catalyst; UCB
Spinal muscular atrophy (SMA) is a hereditary neuromuscular disease that typically begins in infancy or childhood but can manifest at any age.
It is characterized by the irreversible and progressive degeneration of motor neurons in the spinal cord and brainstem. This results in a wide range of symptoms, in addition to which there is substantial variation in the rate of progression and disease prognosis.
Although early diagnosis and timely therapy can slow or prevent disease progression, disease-modifying therapies since 2016 have significantly advanced the management of SMA.
In a clinically focused program, Dr Perry Shieh, a neuromuscular neurologist from the University of California, Los Angeles, discusses the three medications currently approved by the US Food and Drug Administration: nusinersen, risdiplam, and onasemnogene abeparvovec.
He weighs the clinical benefits and key considerations for the use of each drug and emphasizes the need for shared decision-making with the patient.
--
Professor, Departments of Neurology and Pediatrics, University of California, Los Angeles; Neuromuscular Neurologist, Ronald Reagan UCLA Medical Center, Los Angeles, California
Perry Shieh, MD, PhD, has disclosed the following relevant financial relationships:
Serve(d) as a speaker or a member of a speakers bureau for: Grifolis; Biogen; Genentech; CSL Behring; Alexion; Argenx; Catalyst
Received income in an amount equal to or greater than $250 from: Sarepta; Novartis; Biogen; Genentech; Alexion; Argenx; Catalyst; UCB
Spinal muscular atrophy (SMA) is a hereditary neuromuscular disease that typically begins in infancy or childhood but can manifest at any age.
It is characterized by the irreversible and progressive degeneration of motor neurons in the spinal cord and brainstem. This results in a wide range of symptoms, in addition to which there is substantial variation in the rate of progression and disease prognosis.
Although early diagnosis and timely therapy can slow or prevent disease progression, disease-modifying therapies since 2016 have significantly advanced the management of SMA.
In a clinically focused program, Dr Perry Shieh, a neuromuscular neurologist from the University of California, Los Angeles, discusses the three medications currently approved by the US Food and Drug Administration: nusinersen, risdiplam, and onasemnogene abeparvovec.
He weighs the clinical benefits and key considerations for the use of each drug and emphasizes the need for shared decision-making with the patient.
--
Professor, Departments of Neurology and Pediatrics, University of California, Los Angeles; Neuromuscular Neurologist, Ronald Reagan UCLA Medical Center, Los Angeles, California
Perry Shieh, MD, PhD, has disclosed the following relevant financial relationships:
Serve(d) as a speaker or a member of a speakers bureau for: Grifolis; Biogen; Genentech; CSL Behring; Alexion; Argenx; Catalyst
Received income in an amount equal to or greater than $250 from: Sarepta; Novartis; Biogen; Genentech; Alexion; Argenx; Catalyst; UCB
