Sample Portfolio

Welcome to our sample portfolio page. We obviously cannot share the bulk of our work because they are proprietary (intellectual property) assets of the companies and organizations that hired us. We are only able to share publicly available publications. Even with the limited sample portfolio, you will explore some of our extensive range of writing expertise, with a focus on medical writing, clinical trials publications, pre-clinical publications, health economics publications, clinical review papers, and specialized content related to rare diseases. Our dedication to delivering top-notch content is matched only by our commitment to rapid delivery and exceptional customer service, ensuring that we meet and exceed your expectations.

Clinical trials

Finkel RS, Darras BT, Mendell JR, et al. Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG). J Neuromuscul Dis. 2023;10(3):389-404.

Strauss KA, Farrar MA, Muntoni F, et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022;28(7):1381-1389.

Day JW, Mendell JR, Mercuri E, et al. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2022 Feb;45(2):191-192.

Mercuri E, Muntoni F, Baranello G, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(10):832-841.

Al-Zaidy S, Pickard AS, Kotha K, et al. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019;54(2):179-185.

Al-Zaidy SA, Kolb SJ, Lowes L, et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019;6(3):307-317.

Lowes LP, Alfano LN, Arnold WD, et al. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019;98:39-45.

Mendell JR, Al-Zaidy S, Shell R, et al. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med. 2017;377(18):1713-1722.

Clinical reviews

Goodspeed K, Feng C, Laine M, Lund TC. Aspartylglucosaminuria: Clinical Presentation and Potential Therapies. J Child Neurol. 2021;36(5):403-414.

Kabadi AM, Mejia-Guerra, MK, Graef JD, et al. AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities. Curr Opin Biomed Eng. 2024;29:100517.

Mendell JR, Connolly AM, Lehman KJ, et al. Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations. Mol Ther Methods Clin Dev. 2022;25:74-83.

Kichula EA, Proud CM, Farrar MA, et al. Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy. Muscle Nerve. 2021;64(4):413-427.

Petrich J, Marchese D, Jenkins C, Storey M, Blind J. Gene Replacement Therapy: A Primer for the Health-system Pharmacist. J Pharm Pract. 2020;33(6):846-855.

Asher DR, Thapa K, Dharia SD, et al. Clinical development on the frontier: gene therapy for duchenne muscular dystrophy. Expert Opin Biol Ther. 2020;20(3):263-274.

Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019;100:3-11.

Natural history & case studies

Saleh MM, Hamhom AM, Al-Otaibi A, et al. Clinical and Molecular Characteristics of Neuronal Ceroid Lipofuscinosis in Saudi Arabia. Pediatr Neurol. 2024;155:149-155.

Khan TR, Leprince I, Messahel S, Minassian BA, Kayani S. Natural History of SURF1 Deficiency: A Retrospective Chart Review. Pediatr Neurol. 2023;140:40-46.

Retson L, Tiwari N, Vaughn J, et al. Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec. Mol Ther. 2023;31(10):2991-2998.

Health Economics and Outcomes Research (HEOR)

Klimchak AC, Sedita LE, Perfetto EM, Gooch KL, Malone DC. Discriminatory Properties of Quality-Adjusted Life Year Based Cost-Effectiveness Analyses for Patients With Disabilities: A Duchenne Muscular Dystrophy Case Study. Value Health. Published online July 31, 2024. doi:10.1016/j.jval.2024.07.008

Asher D, Dai D, Klimchak AC, Sedita LE, Gooch KL, Rodino-Klapac L. Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec. Mol Ther Methods Clin Dev. 2023;30:474-483.

Klimchak AC, Sedita LE, Rodino-Klapac LR, et al. Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. J Mark Access Health Policy. 2023;11(1):2216518.

Klimchak AC, Sedita LE, Gooch KL, Malone DC. Assessing the impact of single or short-term administration on a therapy's cost-effectiveness: a hypothetical disease-agnostic model. J Med Econ. 2023;26(1):594-602.

Preclinical studies

Potter RA, Peterson EL, Griffin D, et al. Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74. Mol Ther Methods Clin Dev. 2024;32(1):101195.

Potter RA, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy. Biol Open. 2023;12(9):bio059797.

Tukov FF, Mansfield K, Milton M, et al. Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys. Hum Gene Ther. 2022;33(13-14):740-756.

Meseck EK, Guibinga G, Wang S, McElroy C, Hudry E, Mansfield K. Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques. Toxicol Pathol. 2022;50(4):415-431.

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