Amyloidosis Center

Drs. Michelle Kaku and K. H. Vincent Lau conduct both retrospective and prospective research at the Amyloidosis Center. They collaborate with other amyloidosis specialists at the center to examine characteristics of patients with hereditary amyloidosis using a large database of patients with hereditary amyloidosis, supported by a biorepository. They are also currentl sub-investigators in a multicenter clinical trial evaluating the efficacy of a novel treatment for hereditary transthyretin-mediated amyloid polyneuropathy. Dr. Kaku has also studied how treatments for amyloidosis may cross the blood brain barrier in patients with central nervous system involvement of amyloidosis.

Research faculty

Michelle Kaku, MD
K. H. Vincent Lau, MD



Ongoing clinical studies:

“NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy” (NCT04136184)

HELIOS-A, A Phase 3 Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Recent publications:

Lau KHV, Kaku MC, Marks A, Tang C, Zhou L, Siao P. Optimizing the operation of an electrodiagnostic laboratory during the COVID-19 pandemic: A 6-month single-center experience. Muscle Nerve. 2021 Jun 15. doi: 10.1002/mus.27352. Epub ahead of print. PMID: 34131929.

Lau KHV, Hamlyn E, Williams TJ, Qureshi MM, Mak K, Mian A, Cervantes- Arslanian A, Zhu S, Takahashi C. The Effects of Video Instruction on Neuroscience Intensive Care Unit Nursing Skills in Case Presentations and Neurological Examinations. J Neurosci Nurs. 2021 Jun 1;53(3):129-133. doi: 10.1097/JNN.0000000000000591. PMID: 33840806.

Lau KHV, Anand P, Greer DM, Cervantes-Arslanian A, Phicil S, Moore J, Takahashi C. Leveraging Trends in Neurology Admissions for Departmental Planning During the COVID-19 Pandemic. Neurohospitalist. 2021 Apr;11(2):125-130. doi: 10.1177/1941874420960323. Epub 2020 Sep 22. PMID: 33791055

Lau KHV, Kaku MC, Marks A, Tang C, Zhou L, Siao P. Optimizing the operation of an electrodiagnostic laboratory during the COVID-19 pandemic: A 6-month single-center experience. Muscle Nerve. 2021 Jun 15. doi: 10.1002/mus.27352. Epub ahead of print. PMID: 34131929.

Muppidi S, Guptill JT, Jacob S, Li Y, Farrugia ME, Guidon AC, Tavee JO, Kaminski H, Howard JF Jr, Cutter G, Wiendl H, Maas MB, Illa I, Mantegazza R, Murai H, Utsugisawa K, Nowak RJ; CARE-MG Study Group. COVID-19-associated risks and effects in myasthenia gravis (CARE-MG). Lancet Neurol. 2020 Dec;19(12):970-971. doi: 10.1016/S1474-4422(20)30413-0. PMID: 33212055

Anand P, Slama MCC, Kaku M, Ong C, Cervantes-Arslanian AM, Zhou L, David WS, Guidon AC. COVID-19 in patients with myasthenia gravis. Muscle Nerve. 2020 Aug;62(2):254-258. doi: 10.1002/mus.26918. Epub 2020 May 22. PMID: 32392389.

Cervantes-Arslanian A, Lau KHV, Anand P, Shulman JG, Ford T, Takahashi C, Daneshmand A, Nguyen TN, Suchdev K, Abdennadher M, Saxena A, Macone A, Perloff MD, Zhu S, Greer DM. Rapid Dissemination of Protocols for Managing Neurology Inpatients with COVID-19. Ann Neurol. 2020 Aug;88(2):211-214. doi: 10.1002/ana.25774. Epub 2020 Jun 3. PMID: 32395831.

Lau KHV, Kaku M, Bissonnette S, Perloff MD. Three-way telemedicine visits in resident clinic. Med Educ. 2020 Nov;54(11):1061-1062. doi: 10.1111/medu.14331. Epub 2020 Sep 3. PMID: 32885465.

Anand P, Lau KHV, Chung DY, Virmani D, Cervantes-Arslanian AM, Mian AZ, Takahashi CE. Posterior Reversible Encephalopathy Syndrome in Patients with Coronavirus Disease 2019: Two Cases and A Review of The Literature. J Stroke Cerebrovasc Dis. 2020 Nov;29(11):105212. doi: 10.1016/j.jstrokecerebrovasdis.2020.105212. Epub 2020 Jul 30. PMID: 33066908.

Qiu S, Joshi PS, Miller MI, Xue C, Zhou X, Karjadi C, Chang GH, Joshi AS, Dwyer B, Zhu S, Kaku M, Zhou Y, Alderazi YJ, Swaminathan A, Kedar S, Saint- Hilaire MH, Auerbach SH, Yuan J, Sartor EA, Au R, Kolachalama VB. Development and validation of an interpretable deep learning framework for Alzheimer’s disease classification. Brain. 2020 Jun 1;143(6):1920-1933. doi: 10.1093/brain/awaa137. PMID: 32357201.

Kaku M, Siao P. Peripheral Neuropathies. Semin Neurol. 2019 Oct;39(5):517-518. doi: 10.1055/s-0039-1698432. Epub 2019 Oct 22. PMID: 31639834.

Kaku M, Simpson DM. Neuromuscular complications of HIV infection. Handb Clin Neurol. 2018;152:201-212. doi: 10.1016/B978-0-444-63849-6.00016-5. PMID: 29604977.

Kaku M, Berk JL. Neuropathy Associated with Systemic Amyloidosis. Semin Neurol. 2019 Oct;39(5):578-588. doi: 10.1055/s-0039-1688994. Epub 2019 Oct 22. PMID: 31639841.

Siao P, Kaku M. A Clinician’s Approach to Peripheral Neuropathy. Semin Neurol. 2019 Oct;39(5):519-530. doi: 10.1055/s-0039-1694747. Epub 2019 Oct 22. PMID: 31639835.