Publications by authors named "Muna El-Khairi"
Article Synopsis
- - Autoimmune encephalitis (AIE) is a rare neurological disorder characterized by brain inflammation, often linked to specific autoantibodies; currently, there are no approved treatments for AIE despite the involvement of interleukin-6 (IL-6) signaling in its pathology.
- - The CIELO study aims to test the efficacy and safety of satralizumab, an IL-6 receptor-targeting monoclonal antibody, in patients with specific types of AIE, using a randomized, double-blind design with 152 participants.
- - The study will follow a 52-week treatment period with possible extensions, enabling participants to receive either the study drug, placebo, or additional treatment options depending on the
Background: Risdiplam is an orally administered therapy that modifies pre-mRNA splicing of the survival of motor neuron 2 (SMN2) gene and is approved for the treatment of spinal muscular atrophy. The FIREFISH study is investigating the safety and efficacy of risdiplam in treated infants with type 1 spinal muscular atrophy versus historical controls. The primary endpoint of part 2 of the FIREFISH study showed that infants with type 1 spinal muscular atrophy attained the ability to sit without support for at least 5 s after 12 months of treatment.
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- ANCHOVY was a global study examining the natural progression of Type 1 spinal muscular atrophy (SMA) to contextualize findings from the FIREFISH study on risdiplam treatment.
- The research involved analyzing data from 60 patients with SMA symptoms starting between 28 days and 3 months of age, focusing on outcomes like time to death, need for ventilation, and achievement of motor milestones.
- Results showed that patients faced severe challenges, with median ages of around 7.3 months for death or ventilation, and none were able to achieve significant motor skills, highlighting the stark differences compared to those treated with risdiplam in prior studies.
Article Synopsis
- Type 1 spinal muscular atrophy (SMA) is a severe neuromuscular condition that affects infants, characterized by an inability to sit unsupported and low levels of survival of motor neuron (SMN) protein.
- The study tested risdiplam, a medication that enhances SMN protein levels, in infants aged 1 to 7 months and evaluated its effectiveness and safety over 12 months compared to historical controls.
- Results showed that 29% of infants could sit without support after treatment and significant improvements were seen in key motor function assessments versus historical data.
Background: Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam is an orally administered, small molecule that modifies pre-messenger RNA splicing and increases levels of functional SMN protein.
Methods: We report the results of part 1 of a two-part, phase 2-3, open-label study of risdiplam in infants 1 to 7 months of age who had type 1 spinal muscular atrophy, which is characterized by the infant not attaining the ability to sit without support.
In a previous Phase 2 study, olesoxime had a favorable safety profile. Although the primary endpoint was not met, analyses suggested that olesoxime might help in the maintenance of motor function in patients with Types 2/3 SMA. This open-label extension study (OLEOS) further characterizes the safety, tolerability and efficacy of olesoxime over longer therapy durations.
View Article and Find Full Text PDFOutcomes of pancreas transplantation from donors with high alcohol consumption are poorly described. The UK Transplant Registry was used to determine whether donor alcohol intake influenced pancreas survival in simultaneous pancreas-kidney (SPK) transplants performed between 2006 and 2012 (n = 770). Recipients were stratified by donor alcohol intake: group I (n = 122)-high recent alcohol intake (>21 or >14 units of alcohol/week in males or females, respectively) or previous alcohol abuse and group II (n = 648)-low/unknown current intake and no previous alcohol abuse.
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