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FDA halts uniQure’s plans for Huntington’s disease gene therapy
Vinay Prasad, head of the FDA office that regulates gene therapies, has criticized the ways in which the agency has tried to expedite the review of certain genetic medicines. He was also a central figure in the FDA’s request to Sarepta Therapeutics’ to pause distribution of Elevidys, a gene therapy for Duchenne muscular dystrophy.
Summary
AMT-130 is an investigational gene therapy designed to slow Huntington’s disease progression by using a viral vector to deliver genetic material that reduces the production of the toxic huntingtin protein in brain cells. Clinical trial results to date show a significant slowing of disease progression—around 75% reduced progression at 36 months compared to historical controls—along with improvements in cognitive and motor functions, and biomarker evidence suggesting reduced neuronal injury. The therapy involves a one-time neurosurgical delivery and is generally well tolerated. While AMT-130 is specific to Huntington’s disease, the gene therapy platform raises hope for similar approaches in neurodegenerative diseases like Parkinson’s in the future [1], [2], [3].
What is AMT-130?
AMT-130 is the first gene therapy designed to slow the progression of Huntington’s disease (HD), which is caused by a harmful mutation in the huntingtin gene leading to toxic protein accumulation in brain neurons. Developed by uniQure, AMT-130 works by delivering a benign adeno-associated virus (AAV5 vector) carrying a microRNA sequence directly into the brain regions most affected by HD (the caudate nucleus and putamen). This microRNA suppresses the expression of the mutant huntingtin protein, aiming to reduce its toxic effects on neurons [1].
How is AMT-130 administered?
The treatment involves a one-time neurosurgical procedure lasting 12 to 18 hours, where the vector is infused into targeted regions within the brain under real-time imaging guidance. The delivery is permanent, designed to allow long-lasting gene silencing effects without repeated dosing [3].
Clinical trial results and effectiveness
Phase 1/2 trial data involving 29 early-stage HD patients (split into low- and high-dose groups) compared their outcomes to a matched external control group from a large observational study (Enroll-HD). Key findings at 36 months were:
A 75% slowing in progression of the composite Unified Huntington’s Disease Rating Scale (cUHDRS), with the treated group's score declining by −0.38 compared to −1.52 in controls.
A 60% improvement in Total Functional Capacity (TFC), indicating better maintenance of patients' ability to perform daily activities.
Cognitive tests showed improvements with slower decline in processing speed and reading ability.
Biomarkers in cerebrospinal fluid showed an ~8.2% reduction in neurofilament light chain (NfL), a marker of neuronal injury, suggesting less ongoing brain damage.
Early data also suggest reductions in mutant huntingtin protein levels in some patients by about 54%.
Safety profile was generally favorable, with common adverse events related to the surgical procedure including inflammation and headaches, typically manageable [1], [2], [3].
Limitations and critiques of the AMT-130 data
The trial was small (only 29 patients), and only 12 patients per dose group have full 36-month data.
There was no placebo-controlled group; comparisons were made against historical/external matched patients, which can introduce bias.
Results await peer-reviewed journal publication; current information mainly comes from company press releases and secondary reports.
The clinical impact of the numerical scale improvements on day-to-day lives is yet to be fully understood.
The treatment requires invasive brain surgery, which carries risks and limits accessibility.
Cost, availability, and regulatory approval timelines (potentially US launch in 2026–2027) may further restrict widespread use initially [1], [2].
Relevance of AMT-130 to Parkinson's Disease
While AMT-130’s target is mutant huntingtin protein specific to Huntington’s disease, the platform of using gene therapy to reduce toxic protein production is a promising avenue for other neurodegenerative conditions. Experts have suggested that a similar approach might be adapted in the future to target Parkinson’s disease-related proteins such as alpha-synuclein, potentially modifying the course of Parkinson’s. However, this remains theoretical and under research; no approved gene therapy for PD currently exists [3].
Health Disclaimer
Please remember that AMT-130 is currently investigational and not yet approved for general use. Results are preliminary and come from small studies awaiting full peer-reviewed publication. If you or a loved one is affected by Huntington’s disease or Parkinson’s disease and interested in gene therapies or experimental approaches, you should discuss options extensively with your neurologist or movement disorder specialist.
Sources
Gene therapy slows Huntington's disease for first time, clinical trial shows
AMT-130 gene therapy achieves 75% slowing of Huntington's disease progression
Huntington's disease breakthrough: what to know about the gene therapy
Though AMT-130 is focused on Huntington’s disease, its development marks an important milestone in neurodegenerative disease research with exciting prospects for the future of PD treatment. Advances like these bring hope for more targeted and durable therapies.
Huntington’s was the first genetic disease mapped to a specific chromosome. Yet, despite knowing its root cause for more than 40 years, drugmakers have struggled to create effective therapies for the nerve cell-destroying illness. That track record made UniQure’s data all the more exciting. Its trial found that, among 12 participants who were given a high dose of AMT-130 and followed for three years, signs of disease progression appeared to slow by 75%.
Record updated:
11/8/25, 1:54 PM
How gene mutations drive dementia in Parkinson’s disease
New findings suggest that mutations in a gene called GBA – which are a risk factor for developing Parkinson’s disease (PD) – drive cognitive decline by disrupting how neurons communicate with each other in the brain. Patients living with Parkinson’s can experience cognitive symptoms such as difficulty with concentrating and forgetfulness. Over time, many go on to develop dementia.
In the study, researchers analyzed three types of mouse models. Their experiments showed that the SNCA and GBA-SNCA mutants – the two models that had elevated alpha-synuclein – experienced motor deficits that worsened over time, but GBA mutants did not develop any motor deficits. Cognitive deficits, on the other hand, were associated with GBA mutations.
The findings highlight that PD symptoms are driven by different mechanisms, with motor deficits tightly linked to alpha-synuclein buildup and cognitive deficits caused by GBA mutations. While alpha-synuclein aggregations are a common hallmark of Parkinson’s, there is a growing recognition among neuroscientists that not all cases present with this pathology. Click here to learn more.
Record updated:
11/8/25, 1:54 PM
Research head
Parkinson's disease (PD) research aims to understand the causes, mechanisms, and potential treatments for this neurodegenerative disorder.
Areas of Research:
Genetics: Identifying genetic mutations that increase the risk of PD.
Neurobiology: Studying the changes in brain cells and pathways that occur in PD.
Cell Death: Investigating why dopamine-producing neurons die in PD.
Oxidative Stress: Examining the role of oxidative damage in the disease process.
Mitochondrial Dysfunction: Exploring how impaired mitochondrial function contributes to PD.
Stem Cell Therapy: Using stem cells to replace damaged neurons or to protect existing ones.
Gene Therapy: Developing genetic interventions to stop or slow the progression of PD.
Drug Discovery: Identifying and testing new medications to improve symptoms and potentially halt disease progression.
Leading Research Institutions: National Institute of Neurological Disorders and Stroke (NINDS), Parkinson's Foundation, Michael J. Fox Foundation for Parkinson's Research, Harvard Stem Cell Institute, and University of Miami Miller School of Medicine.
Record updated:
11/8/25, 1:54 PM
SwallowFIT, a targeted exercise program designed to retrain the brain’s ability to control the muscles involved in swallowing
A $1.9 million U.S. Department of Defense–funded Phase 2 clinical trial will test SwallowFIT, a targeted exercise program designed to retrain the brain’s ability to control the muscles involved in swallowing — a function often compromised in Parkinson’s disease — in active-duty service members, veterans, or their relatives who have been diagnosed with the disease.
SwallowFIT applies the principles of neuroplasticity (the brain’s ability to adapt and form new connections) to help reorganize the signals sent to the muscles involved in swallowing. Through repeated practice, it strengthens the muscles of the mouth, tongue, and throat while retraining the brain to send clearer signals.
In earlier pilot testing, SwallowFIT helped patients improve their ability to swallow. If the clinical trial succeeds, researchers believe it could pave the way for proactive swallowing therapy to become part of early care for dysphagia. Click here to learn more.
Record updated:
11/13/25, 2:46 PM
Carpet or wood floor for PD
Ensuring home safety is crucial for people with Parkinson’s disease (PD) due to mobility challenges, balance issues, and increased risk of falls. Home improvements focus on reducing fall hazards, improving accessibility, and enhancing lighting and communication. Key modifications include installing grab bars and railings, removing clutter and rugs, securing cords, improving lighting, using stable furniture with armrests, and adding assistive devices like bed rails or shower chairs. Consulting an occupational therapist for a personalized home safety assessment is highly recommended.
https://www.parkinson.org/living-with-parkinsons/management/activities-daily-living/home-safety
https://www.michaeljfox.org/news/7-home-safety-tips-people-parkinsons-disease
Choosing between carpet and wood flooring for a home with Parkinson’s disease (PD) involves balancing safety, comfort, and mobility needs. Carpets, especially low-pile, provide cushioning that reduces injury severity from falls and improves traction, which may help prevent slipping. However, thick or loose rugs can be tripping hazards. Wood floors are easier to clean and accommodate mobility aids like walkers and wheelchairs better, but can be slippery and hard, increasing fall risk and injury severity. Many experts recommend low-pile carpet with secure backing or smooth wood floors treated with non-slip finishes. Personal preference, gait characteristics, and specific mobility challenges guide the best choice.
Carpet Flooring
Advantages:
Low-pile carpets provide cushioning, which can reduce injury severity if a fall occurs.
Carpets with contrasting colors or patterns can provide visual cues to help improve gait and reduce freezing.
Disadvantages:
Thick, uneven, or high-pile carpets increase the risk of tripping or difficulty using mobility aids (walkers, wheelchairs).
Carpets can sometimes “stick” to the feet, which may precipitate freezing of gait.
Recommendations:
Use low-pile, well-secured carpets or rugs with non-slip backing.
Avoid clutter and tripping hazards near carpets.
Use visual cues such as contrasting grout or lines near transitions between flooring types to aid gait.
Wood Flooring
Advantages:
Smooth, flat surface facilitates easier movement with canes, walkers, or wheelchairs.
Easier to clean and maintain than carpet.
Disadvantages:
Can be slippery, increasing fall risk if untreated.
Hard surface may increase injury severity during falls.
Visual pattern changes between tile and wood may trigger freezing of gait in some people.
Recommendations:
Choose hardwood or laminate floors with non-slip finishes.
Avoid highly polished or waxed floors unless treated with anti-slip coatings.
Consider installing flooring with visual cues or subtle texture changes to help step initiation.
Visual Cues and Floor Transitions
Freezing of gait can be triggered by changes in flooring texture or color. Research shows that flooring with horizontal lines or color contrasts can help people with PD step over obstacles and improve gait initiation].
A physical or occupational therapist familiar with Parkinson’s disease can evaluate the home environment and recommend the best flooring based on personal gait characteristics, freezing episodes, and balance status. They can also suggest additional modifications like grab bars, proper lighting, and removal of tripping hazards
Record updated:
1/7/26, 12:48 AM
Common Symptoms of Parkinson’s Disease
The most common Parkinson’s disease symptoms. Remember that although these are the typical symptoms, they can vary greatly from individual to individual—both in terms of their intensity and how they progress. Motor symptoms generally involve movement, while non-motor symptoms do not.
Read about them [HERE]
Record updated:
11/8/25, 1:54 PM
Dystonia (abnormality of muscle tone) v. dyskinesia (abnormality of movement)
A Parkinson's Foundation video of dystonia (abnormality of muscle tone) v. dyskinesia (abnormality of movement) is here.
Key differences between dyskinesia and dystonia are also explained in this HealthCentral story.
Record updated:
11/8/25, 1:54 PM
Mental health symptoms in parkinson’s: common but treatable
https://youtu.be/dzfQi5sfrjU?si=0apNiSAijkq3ndHF
Speaker Bio: Greg Pontone, MD, MHS is Division Chief and Professor Of Aging, Behavioral, and Cognitive Neurology at the University of Florida and Co-Director of Neuropsychiatry Program at The Norman Fixel institute for Neurological Diseases. Dr. Pontone earned his medical degree from the University of South Florida in Tampa. After medical school he completed a medical internship at Johns Hopkins Bayview followed by a residency in psychiatry and a fellowship in geriatric psychiatry and movement disorders research at The Johns Hopkins Hospital in Baltimore, Maryland. Time Stamps:
3:54 – Mental wellness challenges in Parkinson’s: anxiety, depression, apathy, and anger defined and discussed.
4:42 – Breakdown of anxiety symptoms—how it feels mentally and physically, and how it relates to medication cycles.
7:27 – What depression looks like in Parkinson’s and why it’s the single most impactful non-motor symptom.
9:36 – Apathy vs. depression: key differences and why apathy often goes unnoticed or unreported.
11:18 – Why these symptoms happen: neurochemical changes and how Parkinson’s affects mood regulation.
14:57 – Introduction to the “wellness pyramid”: exercise, diet, sleep, and social connection as proactive tools.
22:54 – Strategies for better sleep: sleep hygiene tips, melatonin, and setting a consistent “sleep opportunity.”
25:50 – Socialization’s impact on brain health, mood, and longevity—why isolation is a major risk factor.
33:46 – Medications for depression, anxiety, and apathy: what works, what doesn’t, and why patience is key.
45:25 – How caregivers can help build structure and routine to reduce apathy and support long-term engagement.
54:39 – Dr. Pontone shares what gives him hope: promising research on disease-modifying therapies for Parkinson’s.
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Anxiety in Parkinson’s Disease
Anxiety affects about 40% of people with PD and can manifest as persistent worry, restlessness, panic attacks, physical symptoms (racing heartbeat, sweating, nausea), and avoidance of social situations due to fear or embarrassment.
It can precede motor symptoms or develop during the disease course.
Causes include dopamine loss and changes in other brain chemicals, “off” periods when medication effects wear off, sleep disturbances, social isolation, and uncertainty about the future [2], [4], [2].
Depression in Parkinson’s Disease
Depression affects approximately 30-50% of people with PD.
It is often a direct symptom of PD caused by neurochemical imbalances involving dopamine, serotonin, and norepinephrine, not just a psychological reaction.
Symptoms include persistent sadness, loss of interest, fatigue, feelings of guilt or worthlessness, sleep disturbances, and in severe cases, thoughts of death or suicide.
Depression can worsen motor symptoms and overall functioning but is treatable with medication, therapy, exercise, and social support [1], [3].
Anger and Irritability in Parkinson’s Disease
Anger outbursts and increased irritability are common mood changes in PD, sometimes surprising to families due to personality shifts.
Causes include emotional stress from coping with PD, direct brain changes, medication side effects, cognitive changes, or psychosis.
These behavioral changes often coexist with depression, anxiety, or cognitive decline.
Managing anger involves identifying triggers, medication review, counseling, stress reduction techniques, and caregiver support [5], [2].
Important Considerations
Mental health symptoms may fluctuate with “on/off” medication periods.
Sudden changes in mood or behavior require medical evaluation for infections, medication side effects, or other illnesses.
Non-drug approaches should be tried first when possible.
Safety is paramount if anger or psychosis leads to aggression.
Collaboration among neurologists, psychiatrists, psychologists, and social workers is often needed [3], [2].
Anxiety and Depression in Parkinson’s - Parkinson’s UK Progress Summer
Treatment Approaches To Anxiety and Depression in Parkinson’s Disease
Mood and Cognition Webinar Notes - Stanford Parkinson’s Community
Record updated:
12/12/25, 6:44 PM
Motor-related symptoms
Motor symptoms — which means movement-related symptoms — of Parkinson’s disease include the following:
Slowed movements (bradykinesia).
Tremor while muscles are at rest.
Rigidity or stiffness.
Unstable posture or walking gait.
Additional motor symptoms can include:
Blinking less often than usual.
Cramped or small handwriting.
Mask-like facial expression
Unusually soft speaking voice (hypophonia).
Record updated:
11/8/25, 1:54 PM
Sleep and PD
Sleep Disturbances in Parkinson’s Disease
People with Parkinson’s Disease frequently experience a range of sleep problems that differ from those typical in the general population. Common disturbances include:
Insomnia: Difficulty falling asleep or staying asleep.
REM Sleep Behavior Disorder (RBD): Acting out dreams during REM sleep due to loss of normal muscle paralysis, which can cause injury to self or bed partner.
Restless Legs Syndrome (RLS): Urge to move legs often at night, which disturbs sleep.
Excessive daytime sleepiness: Feeling very sleepy during the day, sometimes to the point of sudden sleep attacks.
Sleep fragmentation: Frequent waking and disrupted sleep architecture.
These disturbances reduce sleep quality, leading to fatigue and worsening other PD symptoms such as cognitive difficulties and mood changes.
Causes and Mechanisms
Sleep dysfunction arises from several PD-related factors:
Neurodegeneration in brainstem and hypothalamic areas critical for sleep regulation.
Effects of dopaminergic and other PD medications can disrupt sleep.
Motor symptoms such as rigidity and tremor interfering with comfortable sleep.
Psychiatric symptoms associated with PD like anxiety and depression exacerbate sleep problems.
Approaches to Improve Sleep in Parkinson’s
Medication Review: Adjustments to PD and other medications can improve sleep quality.
Behavioral Techniques: Sleep hygiene education, regular sleep schedules, reducing daytime naps.
Treatment of Specific Disorders: For example, melatonin or clonazepam for REM sleep behavior disorder.
Light therapy: Shown to improve circadian rhythms and sleep quality in PD.
Exercise: Regular physical activity helps improve sleep and overall motor symptoms.
Addressing co-morbidities: Treating sleep apnea, restless legs, or mood disorders.
Health Disclaimer
Sleep disturbances in people with Parkinson’s Disease are complex and individual. Please consult a neurologist or sleep specialist experienced in PD for diagnostic assessments and tailored treatment plans.
Sources
Record updated:
11/8/25, 1:54 PM
Symptom management - podcast on Improving Quality of Life in Movement Disorders through Non-Motor System Management.
Symptom management By Marco Meglio & Alexa Dessy, MD NeurologyLive.com August 8, 2025 Mind Moments is a podcast from NeurologyLive, and brings you an interview with Alexa Dessy, MD, on Improving Quality of Life in Movement Disorders through Non-Motor System Management. The 17-minute podcast is here.
Record updated:
11/8/25, 1:54 PM
Treatment for Parkinson's Disease
Soon after diagnosis, you will begin exploring the wide range of treatments available to help manage symptoms of Parkinson’s. Since Parkinson’s looks different for everyone, no two treatment paths will be the same. Treatment typically includes some combination of medication, exercise and rehabilitation strategies like physical and speech therapy to manage symptoms and give you the best possible quality of life. Non-medical therapies are just as important as medication. These therapies can treat or prevent many symptoms affecting movement, speech and your ability to do daily life and work activities.
Parkinson's disease is a progressive neurological disorder that affects movement. There is no cure, but treatments can help manage symptoms and improve quality of life.
Medications:
Levodopa: The primary medication for Parkinson's, it increases dopamine levels in the brain.
Dopamine agonists: These medications mimic dopamine and can help reduce tremors and stiffness.
MAO-B inhibitors: These drugs prevent the breakdown of dopamine in the brain.
Anticholinergics: These medications help control tremors and drooling.
Other Therapies:
Deep brain stimulation (DBS): A surgical procedure that implants electrodes in the brain to regulate abnormal electrical activity.
Focused ultrasound surgery (FUS): A non-invasive procedure that uses ultrasound waves to target specific areas of the brain and improve symptoms.
Physical therapy: Exercises to improve balance, coordination, and mobility.
Occupational therapy: Activities to help patients with daily tasks, such as dressing and eating.
Speech therapy: To address speech problems, such as tremors and slurred speech.
Lifestyle changes: Exercise, a healthy diet, and stress management can help improve symptoms.
Emerging Treatments:
Gene therapy: Research is ongoing to develop gene therapies that can stop or slow the progression of Parkinson's.
Stem cell therapy: Stem cells may have the potential to replace damaged dopamine-producing cells in the brain.
Pharmacological interventions: New medications are being developed to target different aspects of Parkinson's pathology.
Personalized Treatment:
The best treatment plan for Parkinson's disease is individualized based on the patient's symptoms, age, and overall health. It's important to work closely with a healthcare team to determine the most effective treatment options.
Record updated:
11/8/25, 1:54 PM
LSVT BIG is a specialized exercise-based therapy designed to improve movement and coordination in people with Parkinson's disease.
What does LSVT BIG do?
Improves balance, gait, and overall mobility
Enhances coordination and agility
Reduces freezing of gait (sudden involuntary stops while walking)
Increases the amplitude (size) of movements
How does LSVT BIG work?
LSVT BIG involves intensive, individualized sessions with a certified therapist. The therapist guides the patient through a series of exercises that progressively challenge their movements, forcing them to make bigger, more exaggerated motions. This helps the brain rewire itself and adapt to the new movement patterns.
Who is LSVT BIG for?
LSVT BIG is primarily intended for individuals with Parkinson's disease who are experiencing movement difficulties. It may also be beneficial for other conditions that affect motor control, such as multiple sclerosis or stroke.
Benefits of LSVT BIG
Studies have shown that LSVT BIG can significantly improve movement and quality of life for people with Parkinson's disease.
It is a safe and well-tolerated therapy.
LSVT BIG can provide long-lasting benefits, even after the therapy sessions are completed.
Where to find LSVT BIG
LSVT BIG is typically offered by physical or occupational therapists who are certified in the program. To find a certified therapist, you can contact your healthcare provider or search online.
Additional Information
LSVT BIG is also known as "Lee Silverman Voice Treatment for Parkinson's Disease Big".
There is also an LSVT LOUD (Lee Silverman Voice Treatment Loud) program, which focuses on improving speech and communication in people with Parkinson's disease.
For more information, visit the LSVT Global website: https://www.lsvtglobal.com/
Record updated:
11/16/25, 10:49 AM
Meditation and Relaxation Techniques
Meditation and Relaxation Techniques
Meditation and relaxation techniques can take many forms. Listening to relaxing music is the most basic form. Mindful meditation can be used to relax and focus on breathing or negative emotions and thoughts. It can also be used to help a person become more aware of their surroundings or body movements. Several studies have shown a connection between Parkinson’s disease symptoms and mindful meditation.