Elevated LDL-cholesterol is a major risk factor for heart disease. In patients with heart disease LDL-cholesterol should be lowered to levels below 70 mg/dl to prevent progression of disease. In most patients life style modification together with lipid lowering drug therapy is sufficient to achieve this goal. In some patients with severe forms of hypercholesterolemia, this may not be sufficient to reach goals and regular lipid apheresis (a costly and time intensive form of therapy) may be performed. Mipomersen is a new drug (apoB antisense oligonucleotide) that can lower LDL-cholesterol even in the most severe forms of LDL-hypercholesterolemia by 25-47%. It is unknown whether and to what extent mipomersen can decrease LDL-cholesterol in patients treated with regular apheresis. Phase 1 of the study will test how 6 months of weekly therapy with mipomersen affects LDL-cholesterol in patients with severe LDL-hypercholesterolemia treated with regular apheresis. Phase 2 will test in how many patients this will result in a meaningful reduction of apheresis time, apheresis frequency or if apheresis can be stopped completely.

Start Date01 Aug 2012

Sponsor / Collaborator

Ludwig-Maximilians-Universität München

NCT01475825

/ CompletedPhase 3

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Followed by an Open-Label Continuation Period to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol

Primary objective:
Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo.
Secondary Objectives:
* Determine whether there are qualitative differences between the safety profiles of the 2 dosing regimens and placebo in Cohort 1, patients with HeFH with LDL-C levels ≥160 mg/dL and <200 mg/dL plus the presence of CHD/risk equivalents (referred to as Cohort 2), and the overall study population
* Determine whether there are qualitative differences between the tolerability of the 2 dosing regimens and placebo in Cohort 1, Cohort 2, and the overall study population
* Further characterize the pharmacokinetics (PK) of the 2 dosing regimens in Cohort 1, Cohort 2, and the overall study population
* Determine whether the 2 mipomersen dosing regimens significantly reduce atherogenic lipid levels in Cohort 2 compared to placebo
* Obtain additional data regarding ongoing safety and efficacy of mipomersen in patients with FH and inadequately controlled LDL-C who complete the primary efficacy assessment visit (PET) in the Blinded Treatment Period and continue treatment in Open-Label Continuation Period

Start Date01 Dec 2011

Sponsor / Collaborator

Kastle Therapeutics LLC

NCT01414881

/ CompletedPhase 1

A Phase 1 Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects

The primary objective of this Phase I exploratory study is to determine the effects of mipomersen on the hepatic production of apolipoprotein-B (apo B) in very low density lipoprotein (VLDL) compared to baseline levels. The study will consist of a Screening Period, a 1-week Run-in Period to establish a stable diet, an approximate 11-week Treatment Period with Placebo or Mipomersen, and a 25-week Post-Treatment Follow-up Period. The total duration of any given subject's participation will be approximately 40 weeks.

Start Date01 Sep 2011

Sponsor / Collaborator

Kastle Therapeutics LLC

[+1]

100 Clinical Results associated with Mipomersen sodium

100 Translational Medicine associated with Mipomersen sodium

100 Patents (Medical) associated with Mipomersen sodium

Literatures (Medical) associated with Mipomersen sodium

01 Jan 2026·POULTRY SCIENCE

Heat stress enhances VLDL secretion in chicken ovarian follicles to potentiate its impact on follicular cell survival and maturation

Article

Author: Sin, Mei-Ying ; Chen, Yu-Hui ; Dettipponpong, Petnamnueng ; Chang, Ling-Chu ; Chen, Shuen-Ei ; Cheng, Chuen-Yu ; Huang, San-Yuan

Chicken ovaries possess the machinery of VLDL production in response to heat stress (HS). The study sought to define the role of VLDL secretion by hierarchical follicles along their development under HS. HS (42°C) for 8 hr (HS8H) or HS3H and following recovery at 37°C to 16 hr (3H13R) upregulated PCNA and/or IL-1β expressions in granulosa (GC) and theca (TH) cells and augmented progesterone (P4) and estradiol (E2) production, respectively. Inhibition of VLDL production by Lomitapide and Mipomersen suppressed P4 secretion at 3H13R and HS8H, but differentially affected E2 production, leading to altered secretion fashions under thermoneutral conditions (37°C, NC) and during post-HS recovery, suggesting interfered steroidogenic competence toward maturation. HS regardless of duration and recovery or interference with VLDL secretion under NC provoked ROS and MDA accumulation in both cell types. GCs transiently increased VLDL secretion at HS3H and showed overt cell death at 3H13R, while TH cells sustained VLDL production and retained viability. Prolonged HS over 8 hr impaired viability of both cell types with disparate responses in lipid dynamics; constantly depressed neutral lipids and cholesterol in GCs, while TH cells showed increased neutral lipids before HS8H but rapid dissipation to HS16H and suppressed cholesterol at HS8H. Surprisingly, Lomitapide and Mipomersen rescued TH cell viability at HS8H and 8H8R in association with alleviated lipid, MDA, and ROS accumulations, whereas GCs exhibited improved viability at HS8H, but not at 8H8R and 3H13R in couple with worse depletion of neutral lipids and cholesterol, suggesting that HS operates at VLDL production to alter cellular lipid dynamics to potentiate cell death, while TH cells are more thermoresistant due to a proficient adaption in lipid disposal. In conclusion, HS enhances VLDL production in hierarchical follicles to augment its impacts on follicular cell fate, but routine secretion of VLDL is obligatory to sustain follicle maturation under normal conditions, in which TH cells are highly agile in lipid remodeling and redox regulation in adaption to heat insults.

31 Dec 2025·ANNALS OF MEDICINE

Unlocking the potential: advancements and applications of gene therapy in severe disorders

Review

Author: M. Elossaily, Gehan ; Makhijani, Shivani ; Ansari, Mohd Nazam ; Ingle, Rahul G.

INTRODUCTION:

Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health.

BACKGROUND:

Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug Administration (FDA) has approved multiple gene therapies such as Kynamro for familial hypercholesterolaemia, Exondys51 for duchenne muscular dystrophy, Spinraza for spinal muscular atrophy, etc., rest for cancer, infectious diseases, and rare diseases.

DISCUSSION:

The authors have summarized recent advances in gene therapy, its background, molecular basis (e.g. viral and non-viral vectors), gene-editing techniques (e.g. CRISPR/Cas9, TALEN, ZFN), and its foremost applications in severe disorders, such as cancer, monogenic disorders (e.g. spinal muscular atrophy), polygenic disorders (e.g. autism), neurogenic disorders (e.g. Parkinson disease and Alzheimer's disease), and infectious diseases (e.g. HIV).

CHALLENGES:

In addition, we explored the major challenges faced by gene therapies during targeted delivery, immunogenicity, efficacy, and safety.

CONCLUSION:

To date, most of the promising approaches, such as different vectors, target cell populations, and both in vivo and ex vivo have paved the foundation for applications of gene therapies. Additionally, advances in enhancing the immune system that would certainly lower the healthcare costs. This review highlights the translatory potential of gene therapy in revolutionizing the treatment landscape for severe disorders.

01 Oct 2025·JOURNAL OF THERMAL BIOLOGY

Functional MTTP and apoB for VLDL secretion in chicken ovaries support small follicle development under heat stress

Article

Author: Sin, Mei-Ying ; Walzem, Rosemary L ; Chen, Yu-Hui ; Chen, Shuen-Ei ; Chang, Ling-Chu ; Dettipponpong, Petnamnueng ; Cheng, Chuen-Yu ; Huang, San-Yuan

By various assessments, the previous study has unequivocally concluded functional apoB and MTTP (microsomal triglyceride transfer protein) for VLDL production in chicken ovaries. The present study sought to use whole tissue culture to define the role of VLDL secretion by small yellow follicles (SYFs) along their development under normal and heat stress (HS) conditions. Under thermoneutral conditions (39 °C), chicken SYFs increased MTTP activity, apoB expression and VLDL secretion, while underwent cell apoptosis along the time course. Despite relieved ER stress and protein ubiquitinylation, inhibition of VLDL secretion by Lomitapide and Mipomersen greatly increased triglyceride accumulation, impaired estradiol production and cell proliferation, and accelerated cell apoptosis in accordance with upregulated caspase 3/7 activity, JNK activation, protein carbonylation, and MDA accumulation. Exposure to HS at 44 °C boosted cell apoptosis in a duration-dependent manner. Acute HS for 3 h enhanced VLDL secretion, impaired estradiol production and cell proliferation, and promoted IL-1b production, oxidative damages, and cell apoptosis, whereas except MDA content and cell proliferation, the detrimental effects were halted after 13 h recovery. Lomitapide and Mipomersen augmented lipid accumulation, oxidative stress, inflammatory response, and exacerbated transient impairment of estradiol secretion and cell proliferation in SYFs under 3 h HS and after recovery, but failed to rescue cell viability despite relieved ER and proteostatic stress. In conclusion, routine secretion of VLDL by SYFs serves as an intrinsic mechanism to sustain cell viability and functions to support the whole program required for follicle development, while under HS, this mechanism provisionally rescues steroidogenesis and cell proliferation.

News (Medical) associated with Mipomersen sodium

08 Jan 2025

·www.pharmaceutical-technology.com

Sanofi taps Alloy Therapeutics for CNS antisense drug development

The companies have teamed up to use Alloy’s AntiClastic Antisense platform. Credit: Bevan Goldswain via Getty Images. Sanofi has entered into a collaboration and license agreement with Alloy Therapeutics to develop an antisense oligonucleotide (ASO) therapy targeting a single, undisclosed central nervous system (CNS) condition. Under the terms of the agreement, Sanofi will pay Alloy up to $27.5m in upfront and preclinical milestone payments, with the potential for more than $400m in additional milestone payments tied to discovery, development, and commercialisation. Alloy is also eligible for tiered royalties on sales of any resulting products. The partnership centres on Alloy’s AntiClastic Antisense platform, which is designed to overcome limitations in existing antisense technologies by targeting RNA-level intracellular disease mechanisms. Alloy claims the platform improves a drug’s therapeutic index and enables delivery of treatments to previously inaccessible targets. Sanofi will leverage its expertise in neuroscience to collaborate on using this platform to develop therapies capable of crossing the blood-brain barrier, a major challenge in central nervous system (CNS) drug development. This collaboration reflects renewed interest in nucleic acid-based therapies, particularly for CNS disorders, an area with both high potential and significant challenges. Notable successes in the field include Biogen’s ASO Spinraza (nusinersen) approved for spinal muscular atrophy. Spinraza generated $1.7bn in sales last year, as per Biogen’s financials. The deal marks Sanofi’s latest investment in ASO research, following its October 2024 investment to Resalis Therapeutics for an ASO therapy targeting obesity pathways. Sanofi has historically explored ASO therapeutics, but one previous effort – Kynamro (mipomersen), developed in partnership with Ionis Pharmaceuticals – faced commercial challenges and was ultimately withdrawn from the market in 2019. Alloy launched its AntiClastic Antisense platform in 2023 after licensing intellectual property from Arnay Sciences. This deal isn’t the first that Alloy has made with big pharma. In November 2024, Alloy teamed up with Takeda to develop Takeda’s proprietary induced pluripotent stem cell (iPSC)-derived chimeric antigen receptor (CAR)-T cell platforms. In May 2024, Alloy licensed its ATX-Gx and ATX-CLC murine platforms to Eli Lilly, providing tools for fully human antibody discovery. These platforms aim to simplify and accelerate therapeutic development. The partnership also integrates Alloy’s technology with Lilly’s Catalyze360 programme, which supports biotech startups by offering drug discovery tools, lab space, and R&D expertise.

License out/inOligonucleotideDrug Approval

11 Dec 2024

·www.echemi.com

Heng Rui Pharma Launches Phase III Trial for ANGPTL3 Monoclonal Antibody

According to information from the U.S. clinical trial registry Clinicaltrials on December 10, Chinese company Heng Rui Pharma has launched its first Phase III clinical trial (SHR-1918-301) for the ANGPTL3 monoclonal antibody SHR-1918. This drug marks the first domestic ANGPTL3 monoclonal antibody to enter the Phase III clinical trial stage. SHR-1918 is an ANGPTL3 monoclonal antibody developed by Heng Rui Pharma, which works by inhibiting ANGPTL3 activity to lower triglycerides (TG) and low-density lipoprotein cholesterol (LDL-C) levels in serum. LDL-C and TG are significant risk factors for the development of atherosclerotic cardiovascular diseases. ANGPTL3 plays a key role in lipid metabolism regulation by inhibiting lipoprotein lipase and endothelial lipase, thereby reducing the clearance of TG and LDL-C. In September this year, Heng Rui Pharma announced the results of its first Phase I study (SHR-1918-101) at the European Society of Cardiology (ESC) annual meeting. The study showed that SHR-1918 (100-1200mg, subcutaneously) demonstrated good tolerance and pharmacokinetic characteristics in healthy subjects, with a peak time of 8.0-10.0 days and a half-life of 29.4-53.5 days. Additionally, SHR-1918 effectively and durably reduced LDL-C and TG levels: patients receiving doses of SHR-1918 of 300mg or higher had LDL-C levels reduced by over 30% (up to 49.1%), lasting more than 64 days; TG levels were reduced by over 50% (up to 82.8%), lasting more than 85 days. Given the potential of SHR-1918 for treating rare dyslipidemia disorders, the drug received breakthrough therapy designation for homozygous familial hypercholesterolemia (HoFH) from the Chinese National Medical Products Administration (NMPA) in September. The launched Phase III study is a multicenter, randomized, double-blind, placebo-controlled clinical trial (n=45), aimed at evaluating the efficacy and safety of SHR-1918 in treating homozygous familial hypercholesterolemia (HoFH). The primary endpoint is the percentage change in LDL-C levels from baseline at week 12. Homozygous familial hypercholesterolemia (HoFH) is a rare and severe genetic disorder where patients accumulate LDL-C in the blood due to defects or deficiencies in low-density lipoprotein receptors (LDLR), increasing the risk of heart disease and stroke. Currently, available treatment options for these patients include statins (simvastatin, atorvastatin, and rosuvastatin), ezetimibe, lomitapide, mipomersen, PCSK9 monoclonal antibodies (evolocumab and alirocumab), and evinacumab. Among these, evinacumab (Regeneron/Ultragenyx) is the only marketed ANGPTL3 monoclonal antibody, with global sales of $80.9 million in 2023.

Phase 3Clinical ResultPhase 1Breakthrough TherapyPhase 2

21 Sep 2023

·synapse.patsnap.com

Medications intervening from the root of difficult and complex diseases: Oligonucleotides

Oligonucleotides (ONs) are a class of drugs composed of single or double strands of oligonucleotides synthesized by artificial chemistry. They operate on mRNA through base complementary pairing, interfering with gene unwinding, replication, transcription, mRNA splicing and processing, output, translation and other stages. They cause abnormally encoded genes to lose function, thereby preventing the expression of "wrong" proteins, and thus exert a unique regulatory mechanism at the gene level on the transcription and translation process of disease genes. Due to the unique chemical structure of oligonucleotide drugs, they exhibit poor drug properties: large molecular weight, strong hydrophilicity, high negative charge, not following Lipinski’s rule. Also, their pharmacokinetic characteristics are poor, they cannot pass through biological membranes, and there might be off-target effects. With the FDA's approval of Mipomersen for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) in 2013 (now discontinued), it was verified that oligonucleotide drugs can be used outside of ophthalmology, which also opened the gene therapy era based on oligonucleotide drugs and brought great opportunities for the development of gene therapy. Some rare and difficult diseases, such as Duchenne Muscular Dystrophy, Hepatitis B and peripheral neuropathy caused by hATTR Amyloidosis, cannot be effectively cured by traditional treatments. However, oligonucleotide drugs can interfere from the root to achieve both symptomatic and radical treatment, increasing medication choices for patients. Compared with traditional protein-targeted drugs, oligonucleotide drugs are more specific and efficient and are expected to fill the treatment field of protein-targeted drugs. With the advancement of technology, the indications have extended to chronic disease treatment, for example, LEQVIO, an siRNA approved by the EMA in 2020 for lipid reduction, requires only two injections per year. In recent years, the continuous development of gene therapy drugs has brought new medication choices for cancer and rare disease patients. The research on oligonucleotide drugs has been going on for more than 30 years. Currently, 15 types of oligonucleotide drugs have been approved for marketing, but there are still issues, such as the modification of the structure of oligonucleotide drugs, optimization of pharmacokinetic characteristics, improvement of delivery systems, enhancement of efficacy, and reduction of side effects, which need continuous exploration. Oligonucleotide Competitive LandscapeAccording to Patsnap Synapse, as of 19 Sep 2023, there are a total of 251 Oligonucleotide drugs worldwide, from 777 organizations, covering 449 targets, 575 indications, and conducting 1780 clinical trials. 👇Please click on the picture link below for free registration or login directly if you have freemium accounts, you can browse the latest research progress on drugs , indications, organizations, clinical trials, clinical results, and drug patents related to this drug type. Approved Oligonucleotide Medicinal: Naltrexone HydrochlorideNaltrexone Hydrochloride is a small molecule drug that targets Toll-like receptor 9 (TLR9). It is primarily used in the treatment of alcohol abstinence, opioid-related disorders, opium dependence, and has also shown potential in the management of COVID-19. The drug was first approved in the United States in November 1984 and is currently approved in both the global and Chinese markets. Naltrexone Hydrochloride is developed by Teva Pharmaceutical Industries Ltd., a prominent player in the pharmaceutical industry. The drug has undergone rigorous testing and has successfully reached the highest phase of development, receiving approval for its therapeutic use. It is classified as an orphan drug, indicating its potential to treat rare diseases or conditions. The therapeutic areas of Naltrexone Hydrochloride extend beyond its primary indications. It has shown promise in the treatment of other diseases, infectious diseases, and respiratory diseases. This versatility highlights the drug's potential to address a wide range of medical conditions. The approval of Naltrexone Hydrochloride in 1984 marked a significant milestone in the pharmaceutical industry. Since then, it has gained recognition for its effectiveness in promoting alcohol abstinence and managing opioid-related disorders. The drug's approval in China further expands its reach and potential impact on a global scale. Naltrexone Hydrochloride has been granted priority review status, indicating its importance in addressing unmet medical needs. This regulatory designation expedites the drug's review process, allowing it to reach patients in need more quickly. In summary, Naltrexone Hydrochloride is a small molecule drug developed by Teva Pharmaceutical Industries Ltd. It targets TLR9 and is primarily used in the treatment of alcohol abstinence, opioid-related disorders, opium dependence, and has potential applications in COVID-19 management. The drug has received approval in the global markets, with its first approval dating back to 1984 in the United States. Its therapeutic areas extend beyond its primary indications, and it has been granted priority review status, emphasizing its significance in addressing medical needs. 👇Please click on the image below to directly access the latest data (R&D Status | Core Patent | Clinical Trial | Approval status in Global countries) of this drug. Approved Oligonucleotide Medicinal: Adjuvanted Hepatitis B vaccineThe Adjuvanted Hepatitis B vaccine is a genetically engineered subunit vaccine that falls under the category of prophylactic vaccines. It is designed to target the HBsAg (Hepatitis B surface antigen) and TLR9 (Toll-like receptor 9). This vaccine is primarily used for the prevention of Hepatitis B, a viral infection that affects the liver. Additionally, it is also indicated for the treatment of kidney failure, chronic. The therapeutic areas in which this vaccine is applicable include urogenital diseases, infectious diseases, and digestive system disorders. Urogenital diseases encompass conditions related to the urinary and reproductive systems, while infectious diseases refer to illnesses caused by pathogenic microorganisms. Digestive system disorders involve ailments affecting the gastrointestinal tract. The Adjuvanted Hepatitis B vaccine (Recombinant) is developed by Dynavax Technologies Corp., a pharmaceutical company specializing in the field of biomedicine. As of the highest phase, this vaccine has received approval, indicating that it has successfully undergone rigorous testing and evaluation to ensure its safety and efficacy. The first approval of this vaccine took place in November 2017 in the United States. This implies that it has met the regulatory requirements set by the U.S. Food and Drug Administration (FDA) for commercial distribution and use within the country. 👇Please click on the image below to directly access the latest data (R&D Status | Core Patent | Clinical Trial | Approval status in Global countries) of this drug. In summary, the Adjuvanted Hepatitis B vaccine (Recombinant) is a genetically engineered subunit vaccine developed by Dynavax Technologies Corp. It is primarily used for the prevention of Hepatitis B and is also indicated for the treatment of kidney failure, chronic. This vaccine targets the HBsAg and TLR9 and falls under the therapeutic areas of urogenital diseases, infectious diseases, and digestive system disorders. Having received approval, it is considered a safe and effective option for immunization against Hepatitis B. The first approval of this vaccine occurred in November 2017 in the United States.

100 Deals associated with Mipomersen sodium

External Link

KEGG	Wiki	ATC	Drug Bank
D08946	Mipomersen sodium	C10AX11	DB05528

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperlipidemia Type IIa	United States	Kastle Therapeutics LLC	29 Jan 2013

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hypercholesterolemia	NDA/BLA	European Union	Genzyme Corp.	-
Heterozygous familial hypercholesterolemia	Phase 3	United States	Kastle Therapeutics LLC	01 Jul 2008
Heterozygous familial hypercholesterolemia	Phase 3	United States	Ionis Pharmaceuticals, Inc.	01 Jul 2008
Heterozygous familial hypercholesterolemia	Phase 3	Canada	Ionis Pharmaceuticals, Inc.	01 Jul 2008
Heterozygous familial hypercholesterolemia	Phase 3	Canada	Kastle Therapeutics LLC	01 Jul 2008
Homozygous familial hypercholesterolemia	Phase 3	United States	Kastle Therapeutics LLC	01 Jul 2007
Homozygous familial hypercholesterolemia	Phase 3	United States	Ionis Pharmaceuticals, Inc.	01 Jul 2007
Homozygous familial hypercholesterolemia	Phase 3	Brazil	Kastle Therapeutics LLC	01 Jul 2007
Homozygous familial hypercholesterolemia	Phase 3	Brazil	Ionis Pharmaceuticals, Inc.	01 Jul 2007
Homozygous familial hypercholesterolemia	Phase 3	Canada	Ionis Pharmaceuticals, Inc.	01 Jul 2007

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01475825 (FOCUS FH, CTgov)	Phase 3	Heterozygous familial hypercholesterolemia \| Hypercholesterolemia \| Hypercholesterolemia, Familial	309	Mipomersen (Regimen A: Mipomersen, 200 mg, Once Weekly)	ooliabovbu(czncfuvfzm) = krlnwhsppg qdhrblxfke (usuiytzyeo, 5.653) View more	-	14 Mar 2019
				Placebo (Regimen A: Placebo, Once Weekly)	ooliabovbu(czncfuvfzm) = tjnukevbaz qdhrblxfke (usuiytzyeo, 6.749) View more
NCT00694109 (CTgov)	Phase 3	Hyperlipoproteinemias \| Hypercholesterolemia, Familial	144	Mipomersen Sodium	plezjfgzsc(fjinmjuyxb) = utmmonmnza nljzpjldpj (xkjbtyvwmv, ecggibmcsx - lkpuxjmivi) View more	-	21 Dec 2015
NCT00694109 (Pubmed \| Eur Heart J) Manual	Phase 3	Hypercholesterolemia, Familial	141	mipomersen	piefciyrns(dblfqgzdaz) = -29% (26-week), -28% (52-week), -30% (76-week) and -31% (104-week) rqjhupapvt (dvfkzpbbqx ) View more	Positive	01 Mar 2015
NCT00607373 \| NCT00794664 \| NCT00770146 \| NCT00706849 (Pubmed \| Arterioscler Thromb Vasc Biol)	Phase 3	Hypercholesterolemia	382	Mipomersen 200 mg	cfpmcocrli(lecbatywje) = hofyrqvlkp twffucxxtm (zlnbxngocd, [ - 42.8, to 5.4]) View more	-	01 Mar 2015
NCT00770146 (Pubmed \| J Am Coll Cardiol) Manual	Phase 3	Hypercholesterolemia	104	Mipomersen	tuotdpnwfy(qkbvywqlmg) = Common adverse events included injection site reactions (78% with mipomersen, 31% with placebo) and flu-like symptoms (34% with mipomersen, 21% with placebo). ljdocyyrao (qcgadnmzdr ) View more	Positive	10 Dec 2013
				Placebo
NCT00477594 (CTgov)	Phase 2	Hyperlipoproteinemias \| Hypercholesterolemia, Familial	21	mipomersen sodium (Mipomersen 200 mg Per Week)	bvgnwltdab(pqamixxose) = qswpmxpcbu xkrpnfwwdz (abtmgntamv, hlyajgxuhc - kgfqxlvmhb) View more	-	05 Apr 2013
				mipomersen sodium (Mipomersen 200 mg Every Other Week)	bvgnwltdab(pqamixxose) = kybkrwzkjq xkrpnfwwdz (abtmgntamv, vxhekxcubs - eqeqfapmlp) View more
NCT00707746 (ASSIST, CTgov)	Phase 2	Hypercholesterolemia	34	placebo (Placebo)	mvcdnfepif(jsajzdqsbg) = fmxwpoefna qwkloivvmf (kfkawempui, 8.40) View more	-	05 Apr 2013
				mipomersen (Mipomersen)	mvcdnfepif(jsajzdqsbg) = ztddfgeirh qwkloivvmf (kfkawempui, 18.43) View more
NCT00706849 (RADICHOL II, CTgov)	Phase 3	Heterozygous familial hypercholesterolemia	124	placebo (Placebo)	gtbjmotndr(sgvzzgewbp) = nsdovdjnvn rwlpbthfff (pltonwstia, 18.02) View more	-	21 Mar 2013
				mipomersen sodium (Mipomersen)	gtbjmotndr(sgvzzgewbp) = fjzamhdikm rwlpbthfff (pltonwstia, 26.99) View more
NCT00607373 (RADICHOL 1, CTgov)	Phase 3	Hyperlipoproteinemias \| Homozygous familial hypercholesterolemia	51	Placebo (Placebo)	rsypjurmuy(xiwjqjwciq) = vsnqzptzit hosywhehke (aeafuoyssi, 17.06) View more	-	21 Mar 2013
				mipomersen (Mipomersen)	rsypjurmuy(xiwjqjwciq) = tzazkmhrkk hosywhehke (aeafuoyssi, 19.85) View more
NCT00794664 (CTgov)	Phase 3	Hypercholesterolemia	58	Placebo (Placebo)	ofwnpxeooi(dfdyswcohs) = trkpeurgbr brmlpsikxt (wstvfrohyd, 46.87) View more	-	21 Mar 2013
				Mipomersen (Mipomersen)	ofwnpxeooi(dfdyswcohs) = nhdtkgywgi brmlpsikxt (wstvfrohyd, 24.71) View more

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