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Acetyl-DL-leucine (ADLL) Treatment Outcomes in MSA Patients

Okay, here’s​ a breakdown of the key information from the provided text regarding each‌ patient, focusing on their symptoms, treatment, and response to ADLL (Tanganil™) ‍therapy. I’ll organize it for clarity.

Vital Note: ⁢ MSA-P+C ⁤refers ⁢to Multiple System Atrophy, Parkinsonian type with Cerebellar features. RBD refers to REM‍ Sleep Behavior Disorder, and pisa ‍syndrome is a lateral bending of the trunk.


patient #1 (Not detailed ⁢enough to summarize ‌effectively – information is missing)

The ⁢text starts to describe Patient #1 but abruptly cuts off. There isn’t enough information to provide a useful summary.


Patient #2

* ⁣ Diagnosis: Parkinson’s Disease with REM Sleep Behavior Disorder (RBD) and Pisa ‌Syndrome.
* Age: ⁢Not specified.
* ⁤ Key Symptoms:

‌ * ⁣ Parkinson’s​ symptoms (general, ⁤not detailed)
⁤ * ‌ Restless ​Legs‍ Syndrome (RLS) ​- initially around age 50, managed ‍with ⁢pramipexole and levodopa.
* REM Sleep Behavior Disorder (RBD) – nightly episodes with falls approximately ⁤once a week, but decreasing in severity over 2-3 years. ⁣he adapted his habitat for safety.
‌ * Pisa Syndrome​ – a leaning of the⁣ trunk.
* Baseline Medication:

* ⁢ Baclofen (for nocturnal cramps)
* ​ Pramipexole⁤ 3.75mg extended-release (for RLS)
‍ * ‌Levodopa 100mg extended-release (for‌ RLS)
* response to ADLL (Tanganil™):

*⁣ Positive: ⁤Nearly complete regression of RBD symptoms within the first four‌ weeks.
* ⁣ Negative: Worsening of balance​ and critically important ⁢ exacerbation of Pisa Syndrome. This necessitated discontinuation of ADLL.
* Rechallenge: Reintroduction of ADLL at a lower dose also caused balance issues,‌ confirming the link.
* Recovery: Balance returned to baseline after stopping ADLL, but RBD symptoms returned.
* Overall: ADLL was⁣ not tolerated due to ⁣its negative impact on balance and Pisa syndrome, despite the benefit for RBD.


Patient ⁣#3

* Diagnosis: Multiple System Atrophy, Parkinsonian type with Cerebellar features (MSA-P+C) ‌- diagnosed 3⁢ years prior, but‌ symptoms⁣ were initially misattributed to psychosomatic causes for 6 ​years.
* Age: ‌46
* key Symptoms:

​ * Erectile Dysfunction (first symptom, age 37)
* ⁤Bladder Dysfunction (requiring catheter, age 40)
⁢ * Extrapyramidal​ Syndrome (progressively disabling, leading to loss of walking ⁤ability)
⁢ * ⁤ Recurrent Infections (pulmonary and urinary,​ exacerbating ⁣MSA symptoms)
‌ ‌ * ⁣ RBD – pronounced early ​on (ages 39-41), but no longer noticeable at the time of ‍the study. He reported non-restorative sleep.
​* Pisa Syndrome – pre-existing.
* Baseline Status: Confined to a wheelchair, institutionalized ​due to infections and worsening MSA.
* Response to ADLL (Tanganil™):

* Initial Effects: Sudden awakenings 3 hours after sleep onset, followed by calm,⁤ deep sleep with fewer dreams.
* ⁣⁣ Negative: ‌ Significant worsening of trunk stability and Pisa Syndrome. Unable to‍ sit upright.‍ Episodes of severe rigidity requiring assistance.
⁣ * Recovery: Symptoms improved to baseline after discontinuation of ADLL,and sleep patterns reverted.
‌ ​* Complication: Developed severe⁣ pulmonary and then urinary infections during the observation period, making it difficult‍ to isolate the effect of⁣ ADLL.
* ⁢ Overall: ​ ADLL ⁢was likely⁣ detrimental,worsening truncal ataxia and rigidity,but the concurrent ​infections complicate the interpretation.


In summary: Both Patient #2 and Patient #3 experienced negative effects on their balance and ​postural ⁣stability (Pisa Syndrome) with ADLL therapy, leading⁣ to its discontinuation. while ADLL showed a ⁤positive effect on RBD in Patient #2, the balance side⁢ effects outweighed the benefit. ⁤ The situation with⁤ Patient #3 is ​more complex due to the complicating factor of infections.

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