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| Name | Class |
|---|---|
| Ipsen | INDUSTRY |
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The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).
Oromandibular dystonia (OMD) is an uncommon, disabling form of cranial dystonia, involving involuntary movements of the lower facial, masticatory, and lingual muscles. This can cause jaw movements including opening, closure, protrusion, retraction, or deviation. Common additional facial movements involve grimacing or lip pursing. When there is tongue involvement, it usually presents as tongue protrusion or curling. Such patients are impaired in relation to eating, speaking and swallowing
This study aims to evaluate the efficacy and safety of a low dose of Dysport® deemed tolerable during phase 1 in subjects with oromandibular dystonia (OMD).
Participants will be injected with Dysport® only, with an unblinded open-label disclosure. The safety and efficacy pf receiving Dysport® will be recorded for all subjects that undergo injection. All subjects will be examined and videotaped at the injection visit, then at 6 and 12 weeks after injection with a standardized protocol. The primary outcome will be blinded examination scores of the videos performed after the study is complete.The evaluators will be three different movement disorders experts, not otherwise involved in the study, who will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). Evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dysport Injections | Experimental | Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Low Dose - AbobotulinumtoxinA | Drug | Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater | This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia | Baseline, Week 6, Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Analogue Pain Scale Score | Measure of jaw pain by visual analogue scale (0-100) where 0 represents "no pain" and 100 represents the "most severe pain". | Baseline, Week 12 |
| Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Stewart A Factor, DO | Emory University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wesley Woods Health Center; Emory University Hospital | Atlanta | Georgia | 30329 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 10599789 | Background | Tan EK, Jankovic J. Botulinum toxin A in patients with oromandibular dystonia: long-term follow-up. Neurology. 1999 Dec 10;53(9):2102-7. doi: 10.1212/wnl.53.9.2102. | |
| 21348790 | Background | Rosales RL, Ng AR, Santos MM, Fernandez HH. The broadening application of chemodenervation in X-linked dystonia-parkinsonism (Part II): an open-label experience with botulinum toxin-A (Dysport(R)) injections for oromandibular, lingual, and truncal-axial dystonias. Int J Neurosci. 2011;121 Suppl 1:44-56. doi: 10.3109/00207454.2011.558260. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dysport Injections | Participants with OMD who have been previously treated with any botulinum toxin Type A were injected with Dysport®. Low Dose - AbobotulinumtoxinA: Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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Participants who received low dose Dysport injections.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dysport Injections | Participants with OMD who have been previously treated with any botulinum toxin Type A were injected with Dysport®. Low Dose - AbobotulinumtoxinA: Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater | This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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Adverse events were collected throughout the duration of the study (4 years).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dysport Injections | Participants with OMD who have been previously treated with any botulinum toxin Type A were injected with Dysport®. Low Dose - AbobotulinumtoxinA: Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dysphagia | Gastrointestinal disorders | Systematic Assessment |
The phase 1 study preceding this trial was intended to assess three different Dysport doses. Two participants developed adverse events at the lowest dose in the phase 1 study. Therefore, the lowest dosage level was used for all phase 2 participants.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Stewart Factor | Emory University | 404-728-4952 | sfactor@emory.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 3, 2015 | Aug 23, 2017 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000071057 | Tardive Dyskinesia |
| ID | Term |
|---|---|
| D004409 | Dyskinesia, Drug-Induced |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C542869 | abobotulinumtoxinA |
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Evaluators will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). The evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.
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The SCS-PD measures drooling. Individual items are scored on a scale from 0-3 where 0 represents "never" and 3 represents "always". The overall maximum score is 21. A higher score indicates greater drooling severity. A lower score indicates lesser severity. |
| Baseline, Week 6, Week 12 |
| Change in Number of Tongue Bites Per Day | The patient will be asked to estimate how many times they tend to accidentally/involuntarily bite their tongue per day. | Baseline, Week 12 |
| Mean Swallowing Disturbance Questionnaire (SDQ-20) Score | Ease of chewing and swallowing will be assessed by the SDQ-20 (modified to exclude question 5 due to redundancy as it relates to drooling and question 15 which is not relevant to the study as it involves prior aspiration pneumonias). Individual items are scored from 0 (never) to 3 (very frequently). The overall score is the total for all items; a higher score indicating more frequent swallowing disturbance; a lower score indicating no or less frequent disturbance, with a possible maximum score of 39. | Baseline, Week 6, Week 12 |
| Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating | The Fahn-Marsden Part B "Speech" Question assesses the ease of producing speech. Responses range from 0=Normal, 1=Slightly involved, easily understood, 2=Some difficulty understanding, 3=Marked difficulty understanding. | Baseline, Week 6, Week 12 |
| Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score | The OMDQ-25 is a subjective quality of life measurement made for patients with Oromandibular Dystonia. The maximum total score is 100 indicating the highest quality of life. A score of 50 indicates a mediocre quality of life. A lower score indicates perceived lower quality of life. | Baseline, Week 6, Week 12 |
| Mean Global Clinical Impression - Improvement Scale (CGI) Index Score | The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. Responses are scored on a scale from 1 to 7; 1 represents "very much improved" and 7 represents "very much worse". | Week 6, Week 12 |
| Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score | The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Responses are scored on a scale from 1 to 7; 1 represents "normal, not at all ill" and 7 represents "among the most extremely ill patients". | Baseline, Week 6, Week 12 |
| Mean Global Clinical Impression- Efficacy Index Score | The Clinical Global Impression - Efficacy Index is a 4×4 rating scale that assesses the therapeutic effect of treatment. Responses range on a scale from 0 to 4 with 4 being the best response. | Week 6, Week 12 |
| Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater | The UDRS measures dystonia severity. The UDRS is being rated by un-blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia. | Baseline, Week 6, Week 12 |
| Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater | This scale measures the severity of dystonia for the jaw and tongue by an un-blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia | Baseline, Week 6, Week 12 |
| Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater | The UDRS measures dystonia severity. The UDRS is being rated by blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia. | Baseline, Week 6, Week 12 |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| Secondary | Change in Analogue Pain Scale Score | Measure of jaw pain by visual analogue scale (0-100) where 0 represents "no pain" and 100 represents the "most severe pain". | Data were not collected. | Posted | Baseline, Week 12 |
|
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| Secondary | Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score | The SCS-PD measures drooling. Individual items are scored on a scale from 0-3 where 0 represents "never" and 3 represents "always". The overall maximum score is 21. A higher score indicates greater drooling severity. A lower score indicates lesser severity. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Change in Number of Tongue Bites Per Day | The patient will be asked to estimate how many times they tend to accidentally/involuntarily bite their tongue per day. | Data were not collected. | Posted | Baseline, Week 12 |
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| Secondary | Mean Swallowing Disturbance Questionnaire (SDQ-20) Score | Ease of chewing and swallowing will be assessed by the SDQ-20 (modified to exclude question 5 due to redundancy as it relates to drooling and question 15 which is not relevant to the study as it involves prior aspiration pneumonias). Individual items are scored from 0 (never) to 3 (very frequently). The overall score is the total for all items; a higher score indicating more frequent swallowing disturbance; a lower score indicating no or less frequent disturbance, with a possible maximum score of 39. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating | The Fahn-Marsden Part B "Speech" Question assesses the ease of producing speech. Responses range from 0=Normal, 1=Slightly involved, easily understood, 2=Some difficulty understanding, 3=Marked difficulty understanding. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score | The OMDQ-25 is a subjective quality of life measurement made for patients with Oromandibular Dystonia. The maximum total score is 100 indicating the highest quality of life. A score of 50 indicates a mediocre quality of life. A lower score indicates perceived lower quality of life. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Global Clinical Impression - Improvement Scale (CGI) Index Score | The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. Responses are scored on a scale from 1 to 7; 1 represents "very much improved" and 7 represents "very much worse". | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Week 6, Week 12 |
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| Secondary | Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score | The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Responses are scored on a scale from 1 to 7; 1 represents "normal, not at all ill" and 7 represents "among the most extremely ill patients". | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Error | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Global Clinical Impression- Efficacy Index Score | The Clinical Global Impression - Efficacy Index is a 4×4 rating scale that assesses the therapeutic effect of treatment. Responses range on a scale from 0 to 4 with 4 being the best response. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Week 6, Week 12 |
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| Secondary | Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater | The UDRS measures dystonia severity. The UDRS is being rated by un-blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater | This scale measures the severity of dystonia for the jaw and tongue by an un-blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| Secondary | Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater | The UDRS measures dystonia severity. The UDRS is being rated by blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia. | Participants who received low dose Dysport injections and completed all study visits. Two participants received two injections each for which data from the second were included in the analysis. For change from baseline to week 6, 18 subjects analyzed, for change from baseline to week 12, 17 subjects analyzed as one subject dropped out at 6 weeks. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 6, Week 12 |
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| 0 |
| 18 |
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| 18 |
| 9 |
| 18 |
| Change in Speech | Nervous system disorders | Systematic Assessment |
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| Soft Palate Weakness | Nervous system disorders | Systematic Assessment |
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| Xerostomia | Metabolism and nutrition disorders | Systematic Assessment |
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| Throat Tightness/Congestion | Gastrointestinal disorders | Systematic Assessment |
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| Drooling | Metabolism and nutrition disorders | Systematic Assessment |
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| Difficulty Chewing | Metabolism and nutrition disorders | Systematic Assessment |
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| Food Falling Out of Mouth | Metabolism and nutrition disorders | Systematic Assessment |
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| Coughing | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Weight Loss | Investigations | Systematic Assessment |
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| Flu-like Symptoms | General disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| D009422 | Nervous System Diseases |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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