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Therapy Area
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New Horizons for Metachromatic Leukodystrophy
7
Mins
March 2025
This article summarises an Orchard Therapeutic-sponsored symposium titled ‘New horizons for metachromatic leukodystrophy – with the advent…
Read more
8
Mins
14 Dec 2023
Spasticity Following Acute Stroke
Many patients experience impairment following a stroke, including motor or cognitive impairment, urinary incontinence, dysphagia…
8
Mins
14 Dec 2023
Tackling Becker Muscular Dystrophy
First described by German physician Peter Emil Becker in the 1950s, Becker is an X-linked genetic recessive disorder. Like Duchenne…
7
Mins
20 Nov 2023
A Review of Oral Fixed-Dose Sodium Phenylbutyrate and Ursodoxicoltaurine in People with Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that is estimated to affect approximately 300,000 individuals worldwide…
11
Mins
4 Oct 2023
Lennox–Gastaut Syndrome: Challenges in Diagnosis – Interviews with Two Key Opinion Leaders
Lennox–Gastaut syndrome (LGS) is a severe childhood-onset epilepsy syndrome that can be challenging to identify due to its heterogeneous and..
13
Mins
14 Sep 2023
The Unspeakable Disease: A Tale of Two Siblings
The deficiency of arylsulfatase A due to the mutations in the ARSA gene is the cause of a rare inherited lysosomal storage disease…
6
Mins
14 Sep 2023
Assessment of Post-Traumatic Stress Disorder in Patients Who Recovered from COVID-19
It is essential to address psychological health, particularly post-traumatic stress disorder (PTSD), among patients who have recovered from…
11
Mins
15 Aug 2023
Focal Onset Seizures: New Treatment Options in The Clinical Practice
Carreño began by discussing the spectrum of uncontrolled epilepsies. They stressed that uncontrolled epilepsy does not necessarily…
15 Aug 2023
Retracted: A Spotlight on Friedreich Ataxia: Optimising the Patient Journey from Diagnosis to Disease Management
This article has now been retracted and a retraction statement has been published.
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