A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Subjects in the randomized study arms will continue to take the standard of care (SOC), lonafarnib, and will be randomized to either take SOC alone or in combination with progerinin.

Start Date13 Jan 2025

Sponsor / Collaborator

PRG S&T Co., Ltd

NCT05953545

/ WithdrawnPhase 2IIT

Open Label Phase 2 Clinical Study of Peginterferon Lambda and Lonafarnib Boosted With Ritonavir 48-Week Combination Therapy for Delta Hepatitis

Background:
Chronic hepatitis D is a serious liver disease caused by a virus. Currently, no medications are approved to treat chronic hepatitis D.
Objective:
To test a combination of 3 drugs in people with chronic hepatitis D.
Eligibility:
People 18 years or older with chronic hepatitis D.
Design:
Participants will be in the study about 2 years. They will have 3 inpatient stays of 3 to 5 days.
Participants will be screened. They will have a physical exam with blood tests. They will have a test of their heart function and an ultrasound: a wand that uses sound waves to create images of the liver will be rubbed over the skin on their torso.
Participants will stay in the clinic for a 3-day baseline visit. They will have imaging scans, an eye exam, and a visit with a reproductive specialist. They will have a liver biopsy: about 1 inch of liver tissue will be removed, either with a tube inserted through a vein in the neck, or with a needle inserted through the participant s side.
Participants will take the study drugs for 48 weeks. Two of them are tablets taken twice a day at home; 1 is a shot administered once a week. Participants will begin taking the drugs during a 5-day stay in the clinic. Then they will have 15 outpatient visits while taking the drugs and 7 more after they finish.
The last 3-day clinic stay will be 6 months after participants finish taking the drugs. The liver biopsy, imaging scans, and other tests will be repeated.

Start Date22 May 2024

Sponsor / Collaborator

National Institute of Diabetes & Digestive & Kidney Diseases

ChiCTR2100050052

/ Not yet recruitingNot ApplicableIIT

Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy

Start Date18 Aug 2021

Sponsor / Collaborator

The Children's Hospital of Zhejiang University School of Medicine

100 Clinical Results associated with Lonafarnib

100 Translational Medicine associated with Lonafarnib

100 Patents (Medical) associated with Lonafarnib

305

Literatures (Medical) associated with Lonafarnib

01 Dec 2026·MECHANISMS OF AGEING AND DEVELOPMENT

Hierarchical homeostatic failure in Hutchinson–Gilford progeria syndrome: From nuclear lesion to systemic decline

Article

Author: Djabali, Karima

Hutchinson-Gilford progeria syndrome (HGPS) is a fatal laminopathy caused by an LMNA mutation that generates progerin, a permanently farnesylated, truncated form of prelamin A. Although progerin's structural effects are well established, prior reviews have largely cataloged its downstream consequences, genome instability, proteostasis failure, inflammation, stem-cell dysfunction and matrix remodeling, as parallel, largely independent processes. Here, two decades of mechanistic evidence are reorganized into a single evidence-ranked hierarchy spanning four interconnected levels of homeostasis, nuclear, cellular, tissue and organismal, that explicitly separates established mechanisms from inferred cross-scale links. Progerin perturbs post-mitotic nuclear reassembly, but several defining abnormalities emerge or intensify during interphase and across successive cell generations, indicating progressive maintenance failure rather than a purely static structural model. Nuclear dysfunction is associated with impaired proteostasis, stress adaptation and lineage competence; these cellular defects can be amplified by extracellular-matrix remodeling, chronic inflammation, and reduced regenerative capacity. Evidence is strongest for nuclear and cellular mechanisms, whereas a continuous nucleus-to-organism sequence remains an integrative, testable model. When fragmented mechanisms are ordered across biological scales, this framework helps explain disease latency, tissue selectivity, partial reversibility, and the rationale for combination therapy. Lonafarnib, proteostasis-directed interventions, JAK-STAT inhibition and genome-, RNA-, and progerin-directed strategies are therefore considered complementary approaches acting at distinct levels of the hierarchy. HGPS is not a replica of physiological aging, but a genetically defined system that reveals how persistent nuclear stress can progressively erode conserved adaptive networks.

01 Nov 2026·PHARMACOLOGICAL RESEARCH

MT-Exo-derived miR-4651 and miR-6126 coordinately regulate the SRSF1–progerin and SP1–PrPᶜ axes to attenuate vascular aging in HGPS

Article

Author: Kim, Su-Jin ; Lee, Jaeyoung ; Song, Yun Seob ; Lee, Sang Hun ; Jeong, Kyung Hee ; Park, Ji Sun ; Jeon, Myung Shin ; Lee, Ji Eun ; Joo, Eun Young ; Choi, Gwang-Seong ; Yoon, Unyoung

Hutchinson-Gilford progeria syndrome (HGPS) is characterized by progressive vascular injury that remains incompletely controlled by therapies targeting progerin-associated pathology. Here, we investigated the therapeutic activity and molecular mechanisms of melatonin-preconditioned mesenchymal stromal cell-derived small extracellular vesicles (MT-Exo) using a patient-anchored translational framework integrating longitudinal vascular phenotyping and serial serum samples from an index patient with HGPS with independently sourced HGPS fibroblasts and endothelial-cell models. Serial HGPS serum samples exhibited increased progerin and cellular prion protein (PrPᶜ) signals, together with elevated troponin I, in the setting of progressive multisite vasculopathy. HGPS serum impaired endothelial morphology and angiogenic network formation, whereas recombinant PrPᶜ induced cellular-stress, inflammatory, chromatin, and nuclear-lamina abnormalities. MT-Exo attenuated HGPS serum-induced endothelial dysfunction and reduced progerin, PrPᶜ, p16, and γH2AX abnormalities and lamin A/C disorganization in HGPS fibroblasts. Mechanistically, MT-Exo-enriched miR-4651 directly targeted SRSF1 and attenuated progerin-associated nuclear-lamina abnormalities, whereas MT-Exo-enriched miR-6126 directly targeted SP1 and reduced PRNP/PrPᶜ expression. These two microRNAs therefore acted through distinct but complementary regulatory pathways involving SRSF1-progerin and SP1-PRNP/PrPᶜ signaling. A prespecified patient-informed virtual-cohort analysis of 15 treatment strategies further prioritized a cluster of lonafarnib-based combinations containing MT-Exo, dual miRNA, or miR-6126 for subsequent experimental evaluation, although no single regimen was consistently top-ranked across models. Collectively, these findings identify complementary miR-4651-SRSF1-progerin and miR-6126-SP1-PRNP/PrPᶜ regulatory axes in HGPS vascular aging and provide a mechanistic rationale for preclinical evaluation of MT-Exo- and miRNA-based combination strategies.

01 Nov 2026·JOURNAL OF PLANT PHYSIOLOGY

Thirty years of plant farnesylation research: Few substrates, many gaps, and a clear path forward

Review

Author: Sundaresan, Anjana P ; Distefano, Mark D ; Daszkowska-Golec, Agata

Protein farnesylation - the covalent attachment of a 15-carbon farnesyl isoprenoid to a C-terminal CaaX motif by protein farnesyltransferase (PFT, FTase) - is one of the most functionally important post-translational modifications in eukaryotic biology. In mammals, the biomedical significance of farnesylation is well established. The FTase inhibitor (FTI) lonafarnib treats Hutchinson-Gilford progeria syndrome. FTase remains an oncology target, and chemical proteomic strategies have mapped more than 100 farnesylated substrates at single-protein resolution. In plants, genetic evidence establishes that FTase governs abscisic acid (ABA) signalling, brassinosteroid (BR) homeostasis, cytokinin (CK) partitioning, meristem development, thermotolerance, innate immunity, nitrogen sensing, and drought resilience across Arabidopsis thaliana, barley (Hordeum vulgare), canola (Brassica napus), wheat (Triticum aestivum), soybean (Glycine max), and rice (Oryza sativa). Yet fewer than fifteen substrates have been biochemically confirmed in any plant species after nearly three decades of research, and no crop species has been subjected to prenylome-scale chemical proteomics. This review synthesizes the plant and animal farnesylation literature and argues that this disconnect is methodological rather than biological. We trace the arc from FTase enzymology through the emerging catalogue of confirmed plant substrates, exemplified by the HSP40 co-chaperones J2/J3, the cytokinin-biosynthetic enzyme AtIPT3, and plasmodesmal HIPPs, to crop engineering outcomes. We propose a spatial-switch model to organize the confirmed substrate data into a unifying mechanistic framework, while acknowledging that this model awaits systematic experimental testing. Finally, we outline the chemical biology tools - alkyne-isoprenoid probes, quantitative prenylomics profiling - that must be adapted for plant systems to close the gap between substrate prediction and biochemical validation. Farnesylation exemplifies how a conserved eukaryotic modification can be harnessed for crop improvement once the underlying substrate biology is sufficiently understood.

News (Medical) associated with Lonafarnib

27 Oct 2026

·www.biospace.com

EIT Pharma Appoints Dr. Joseph Toerner, M.D., M.P.H., as Chief Medical Officer

KIRKLAND, Wash. , Oct. 27, 2026 /PRNewswire/ -- EIT Pharma, Inc. ("EIT Pharma" or the "Company"), a biotechnology company focused on developing therapies for infectious diseases, today announced the appointment of Dr. Joseph Toerner, M.D., M.P.H., as Chief Medical Officer, effective immediately. Dr. Toerner brings 26 years of experience at the U.S. Food and Drug Administration (FDA), including leadership roles in hepatology, infectious diseases, and drug safety. As Chief Medical Officer, Dr. Toerner will provide medical and clinical leadership across EIT Pharma's development programs, including supporting the ongoing regulatory review of lonafarnib, its late-stage investigational first-in-class oral therapy for chronic hepatitis D (CHD), and peginterferon lambda, its Phase 2 investigational treatment for severe acute viral respiratory infections. "Dr. Toerner brings a rare combination of an infectious disease physician's perspective with more than two decades at the FDA, and few understand the path from clinical evidence to approved therapy as deeply as he does," said Dr. Leen Kawas, PhD, Chief Executive Officer of EIT Pharma. "We are delighted he has joined our team and his expertise will strengthen the clinical and medical foundation of our work as we advance our infectious disease pipeline and progress therapies toward the patients waiting for them." "People living with chronic hepatitis D have far too few treatment options, and I share EIT Pharma's conviction that no patient should be left behind," said Dr. Toerner. "My career has been defined by work where the unmet need is high and the science is rigorous, and EIT Pharma's programs reflect both. I am honored to join EIT Pharma and look forward to working with Dr. Kawas and the team to advance lonafarnib and lambda for patients." Dr. Toerner held several leadership positions at the FDA, most recently serving as Director of the Division of Hepatology and Nutrition from 2020 to 2024. His prior roles spanned antiviral drug development, vaccines, drug safety, and regulatory policy. Since leaving the FDA in 2024, he established Joseph Toerner, LLC to provide consulting services in drug development, including hepatology, nutrition, vaccines, and infectious diseases. Dr. Toerner received his B.S. from the University of Dayton, his M.D. from Case Western Reserve University School of Medicine, and his M.P.H. from Johns Hopkins Bloomberg School of Public Health. He completed his residency in internal medicine at Case Western and his fellowship in infectious disease at Georgetown University Medical Center. He holds Board Certifications in Internal Medicine and Infectious Diseases. About EIT Pharma, Inc. EIT Pharma is a biotechnology company focused on advancing therapies for infectious diseases with significant unmet medical needs. The company is developing a portfolio of programs designed to address serious infectious diseases through rigorous science, disciplined execution, and a patient-centered approach focused on translating scientific innovation into meaningful real-world impact for patients. About Lonafarnib Lonafarnib is an investigational oral therapy candidate being evaluated for the treatment of chronic hepatitis D. Lonafarnib is designed to target a key step in the hepatitis D virus lifecycle and is currently the only oral therapeutic candidate in late-stage clinical development for chronic hepatitis D. Lonafarnib has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for the treatment of chronic hepatitis D. The safety and efficacy of lonafarnib have not been established. About Peginterferon Lambda-1a Peginterferon lambda-1a is an investigational therapy candidate being evaluated for the treatment of Acute Respiratory Infection. Peginterferon lambda-1a is designed to act as an antiviral to enhance innate immunity by inducing interferon-stimulated genes in immune cells. Peginterferon lambda-1a has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for the treatment of Acute Respiratory Infection. The safety and efficacy of peginterferon lambda-1a have not been established. Forward-Looking Statements This press release contains forward-looking statements regarding regulatory review timelines, anticipated milestones, potential product characteristics, and future development plans. These statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. Lonafarnib is an investigational product candidate and has not been approved by the FDA. There can be no assurance that the FDA will approve the NDA or that the product will become commercially available. EIT Pharma undertakes no obligation to update forward-looking statements except as required by law. Media Contact: Reevemark Jacqueline Zuhse/Jamie Olensky EITPharmaTeam@reevemark.com 212-433-4600 View original content to download multimedia: SOURCE EIT Pharma

Phase 2Executive ChangeDrug Approval

20 Oct 2026

·www.prnewswire.com

EIT Pharma Presents New Lonafarnib Analyses at the 2026 Delta Cure International Meeting

FDA Previously Accepted the New Drug Application for Lonafarnib, an Investigational First-in-Class Oral Therapy for Chronic Hepatitis D KIRKLAND, Wash., Oct. 20, 2026 /PRNewswire/ -- EIT Pharma, Inc. ("EIT Pharma" or the "Company"), a biotechnology company focused on developing therapies for infectious diseases, today highlighted three presentations at the 5th Edition of the Delta Cure International Meeting in Milan, Italy. The Company presented new analyses from its lonafarnib development program for Chronic Hepatitis Delta (CHD), covering the durability of response after treatment ends, histologic improvement and the clinical significance of alanine aminotransferase (ALT) dynamics following treatment cessation. Lonafarnib is an investigational first-in-class oral CHD therapy candidate with a unique mechanism of action. The New Drug Application for Lonafarnib for the treatment of CHD is under review by the U.S. Food and Drug Administration. "Delta Cure has become one of the most important forums bringing together the global hepatitis D community, and we are proud to support this year's meeting and share new analyses from the lonafarnib development program," said Dr. Leen Kawas, Chief Executive Officer of EIT Pharma. "The findings presented at Delta Cure explore important questions regarding durability of response, liver health, and clinical outcomes following treatment and we believe continued research in these areas is essential as the hepatitis D community works toward better solutions for patients." Key findings from each of the presentations include: Histologic improvement and reduction in necroinflammation with lonafarnib-based therapy in chronic hepatitis delta: phase 3 D-LIVR trial • Presented by Dr. Nancy Reau • Read the selected Oral poster presentation here Post-treatment response following 48 weeks of lonafarnib therapy: analysis of 24-week follow-up in the phase 3 D-LIVR trial in chronic hepatitis delta • Presented by Dr. Saeed Hamid • Read the poster here Clinical significance of ALT dynamics following lonafarnib treatment cessation in the phase 3 D-LIVR trial • Presented by Dr. Ohad Etzion • Read the poster here About Lonafarnib Lonafarnib is an investigational oral therapy candidate being evaluated for the treatment of chronic hepatitis D. Lonafarnib is designed to target a key step in the hepatitis D virus lifecycle and is currently the only oral therapeutic candidate in late-stage clinical development for chronic hepatitis D. Lonafarnib has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for the treatment of chronic hepatitis D. The safety and efficacy of lonafarnib have not been established. About EIT Pharma, Inc. EIT Pharma is a biotechnology company focused on advancing therapies for infectious diseases with significant unmet medical needs. The Company is developing a portfolio of programs designed to address serious infectious diseases through rigorous science, disciplined execution, and a patient-centered approach focused on translating scientific innovation into meaningful real-world impact for patients. Forward-Looking Statements This press release contains forward-looking statements regarding regulatory review timelines, anticipated milestones, potential product characteristics, and future development plans. These statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. Lonafarnib is an investigational product candidate and has not been approved by the FDA. There can be no assurance that the FDA will approve the NDA or that the product will become commercially available. EIT undertakes no obligation to update forward-looking statements except as required by law. Media Contact Reevemark Jacqueline Zuhse/Jill Steinman [email protected] 212-433-4600 SOURCE EIT Pharma 21% more press release views with Request a Demo

Phase 3Clinical ResultDrug ApprovalNDA

13 Oct 2026

·www.prnewswire.com

EIT Pharma to Sponsor and Present at the 2026 Delta Cure International Meeting

KIRKLAND, Wash., Oct. 13, 2026 /PRNewswire/ -- EIT Pharma, Inc. ("EIT Pharma" or the "Company"), a biotechnology company focused on developing therapies for infectious diseases, today announced it will sponsor and present at the 5th Edition of the Delta Cure International Meeting to be held on October 15-16, 2026, in Milan, Italy. Delta Cure is recognized as the leading international forum dedicated to Hepatitis Delta Virus and its programming features a comprehensive exploration of the virus, epidemiology, virology, immunology, clinical progression, and effect of therapy on long-term complications. EIT Pharma will present new analyses from the lonafarnib development program for Chronic Hepatitis Delta (CHD). Lonafarnib is currently the only late-stage investigational therapy for CHD with an oral route of administration. EIT Pharma's Delta Cure International Meeting 2026 Presentation Details: Oral Presentation: Title: Histologic improvement and reduction in necroinflammation with lonafarnib-based therapy in chronic Hepatitis Delta: phase 3 D-LIVR trial Presenter: Dr. Nancy Reau, Associate Director of Solid Organ Transplantation and Section Chief of Hepatology at Rush University Medical Center Date: Friday, October 16, 2026, 12:00pm CEST Poster: 104 Breakfast Briefing: Title: Sunrise on Lonafarnib: EIT Pharma Breakfast Briefing Date: Friday, October 16, 2026, 7:00am CEST Poster Presentations: Poster 105: Post-treatment response following 48 weeks of lonafarnib therapy: analysis of 24-week follow-up in the phase 3 D-LIVR trial in chronic hepatitis delta; presented by Dr. Saeed Hamid Poster 106: Clinical significance of ALT dynamics following lonafarnib treatment cessation in the phase 3 D-LIVR trial; presented by Dr. Ohad Etzion Earlier this year, the U.S. Food and Drug Administration (FDA) accepted the New Drug Application for lonafarnib for the treatment of CHD, a serious life-threatening liver disease caused by hepatitis D virus in individuals who already have hepatitis B virus infection. About Lonafarnib Lonafarnib is an investigational oral therapy candidate being evaluated for the treatment of chronic hepatitis D. Lonafarnib is designed to target a key step in the hepatitis D virus lifecycle and is currently the only oral therapeutic candidate in late-stage clinical development for chronic hepatitis D. Lonafarnib has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for the treatment of chronic hepatitis D. The safety and efficacy of lonafarnib have not been established. About EIT Pharma, Inc. EIT Pharma is a biotechnology company focused on advancing therapies for infectious diseases with significant unmet medical needs. The company is developing a portfolio of programs designed to address serious infectious diseases through rigorous science, disciplined execution, and a patient-centered approach focused on translating scientific innovation into meaningful real-world impact for patients. Forward-Looking Statements This press release contains forward-looking statements regarding regulatory review timelines, anticipated milestones, potential product characteristics, and future development plans. These statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. Lonafarnib is an investigational product candidate and has not been approved by the FDA. There can be no assurance that the FDA will approve the NDA or that the product will become commercially available. EIT Pharma undertakes no obligation to update forward-looking statements except as required by law. Media Contact Reevemark Jacqueline Zuhse/Jill Steinman [email protected] 212-433-4600 SOURCE EIT Pharma 21% more press release views with Request a Demo

Phase 3Drug Approval

100 Deals associated with Lonafarnib

External Link

KEGG	Wiki	ATC	Drug Bank
D04768	Lonafarnib	A16AX20 A16AX	DB06448

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Laminopathies	European Union	-	18 Jul 2022
Laminopathies	Iceland	-	18 Jul 2022
Laminopathies	Liechtenstein	-	18 Jul 2022
Laminopathies	Norway	-	18 Jul 2022
Hutchinson-Gilford progeria syndrome	United States	Sentynl Therapeutics, Inc.	20 Nov 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hepatitis D, Chronic	Phase 3	Israel	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D, Chronic	Phase 3	New Zealand	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D, Chronic	Phase 3	Turkey	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D	Phase 3	United States	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Belgium	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Bulgaria	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Canada	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	France	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Germany	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Greece	Eiger BioPharmaceuticals, Inc.	01 Dec 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03719313 (D-LIVR, AASLD2024)	Phase 3	Hepatitis D	88	Pegylated-interferon-α-2a (pegIFN) + Ritonavir-boosted lonafarnib (LNF)	tuctejilck(cdcdevfoug) = dyfnebouzk kpqgajtywl (agatpkfhmt ) View more	Positive	15 Nov 2024
NCT02527707 (LOWR-4, CTgov)	Phase 2	Hepatitis D, Chronic	15	lonafarnib+Ritonavir	fjseftkxlu(oniiwzcywp) = ubpktqvunt txksmlsuxy (xpeghbrpcl, 1.4) View more	-	22 Jun 2023
NCT00425607 \| NCT00916747 (Pubmed \| Genet Med)	Not Applicable	Laminopathies \| Hutchinson-Gilford progeria syndrome	-	Lonafarnib	lkiujhzbvd(envhnhbscp) = ynoohblgrz bscrujfpif (uaxagrhwfp )	Positive	12 Dec 2022
NCT02430181 (LOWR-1, CTgov)	Phase 2	Hepatitis D, Chronic	21	lonafarnib+Ritonavir	eupfqusbiq(stsfrtdnhz) = bvvokvcczl wrjdftwkfr (vrsunwmbgu, yzfjtrwqrp - oxcnffmkmp) View more	-	29 Nov 2022
NCT03600714 (CTgov)	Phase 2	Hepatitis D, Chronic	26	Lonafarnib+Peg-interferon lambda+ritonavir	mvbxkboyll = jangzxfmnj skjhiytfze (ldozumzqzm, hrgexwcpfp - vzhzakwszr) View more	-	01 Dec 2021
LIFT HDV Study (EASL2020)	Phase 2	Hepatitis D HDV RNA \| HBV DNA	26	Peginterferon Lambda	ahzdsnbwnh(hokbvxtwgm) = lcsdbqfjvs hlwrctuemz (uozhcaqnmg, 2.9–3.8)	-	27 Aug 2020
LIFT HDV Study (EASL2020)	Phase 2	Hepatitis D HDV RNA \| HBV DNA	26	Lonafarnib	ahzdsnbwnh(hokbvxtwgm) = cvaoyvxiaw hlwrctuemz (uozhcaqnmg, 2.9–4.5)	-	27 Aug 2020
Pubmed Manual	Not Applicable	Hutchinson-Gilford progeria syndrome	258	Lonafarnib	emrozmfzfx(biuliupyaq) = Treatment was associated with a lower mortality rate (hazard ratio, 0.12; 95% CI, 0.01-0.93; P = .04). In the combined cohort, there were 4 deaths (6.3%) among 63 patients in the treated group and 17 deaths (27.0%) among 63 patients in the matched untreated group (hazard ratio, 0.23; 95% CI, 0.06-0.90; P = .04). mnordqottr (vfxbjycufs )	Positive	24 Apr 2018
Pubmed Manual	Not Applicable	Hutchinson-Gilford progeria syndrome	258	control		Positive	24 Apr 2018
NCT00879034 (CTgov)	Phase 2	Laminopathies \| Hutchinson-Gilford progeria syndrome	5	Lonafarnib+Pravastatin+Zoledronic Acid	sxgkybfigk = oytrdlkhxb wwvempcepg (ljuoeikthg, orwmafxiqe - gcuvsedbqe) View more	-	31 Jul 2017
NCT00425607 (CTgov)	Phase 2	Laminopathies \| Hutchinson-Gilford progeria syndrome	29	Lonafarnib	bjwnfeimzg = cgwsnwnacd jvfpbqacmh (oftpkknqdd, eunoyibfwb - sxhlhqudse) View more	-	24 May 2017

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