<h2>Rare but not so rare</h2>
Although considered a rare disease in the general population, the prevalence of Gaucher disease is significantly higher in specific patient groups.1
Think Gaucher disease when considering the following:
<ul>
 <li>
 Patients presenting with splenomegaly and/or thrombocytopenia</li>
 <li>
 Patients with family members affected by Gaucher disease</li>
 <li>
 Patients of Ashkenazi Jewish descent</li>
</ul>
Although the disease can affect anyone of any ethnicity, there is a high prevalence of Gaucher disease in certain ethnic backgrounds. Notably those of Ashkenazi Jewish descent have a disease prevalence of approximately 1 in 800.2
Approximately 1 in every 15 people of Ashkenazi descent is a carrier of Type 1 Gaucher disease.1 Therefore it is important to test close family of patients from this background.

As an autosomal recessive disorder, a carrier of Gaucher disease has a 50% chance of having a child which carries the Gaucher gene. If both parents are carriers, there is a 25% chance that the child will be affected with Gaucher disease. If your patient has an immediate relative who has been diagnosed with Gaucher disease, it is important to order a blood test straight away.

GAUCHER

RARE-DISEASE

Epidemiology of Gaucher disease

<h2>What to look out for</h2>
The signs of Gaucher disease may look similar to other haematological malignancies. Rule out Gaucher disease in patients who present with these symptoms.
Four key symptoms to look out for are:1
<ul>
 <li>
 Splenomegaly</li>
 <li>
 Thrombocytopenia</li>
 <li>
 Hepatomegaly</li>
 <li>
 Bone pain</li>
</ul>

<h2>What to look out for</h2>
The journey to diagnosis can be a challenge. However, there are some key symptoms and signs to look out for:2,3

Gaucher symptoms prevalence data from Charrow J et al. Arch Intern Med. 2000. 160:2835–2843.3

When considering patients with these symptoms, where malignancy has been ruled out, think Gaucher disease.3–20

Signs and Symptoms of Gaucher disease

This is intended for HCPs practising in Great Britain (England, Scotland, Wales) only.
Watch the video below to find out how our 30-year commitment to rare disease1&nbsp;means we always lead with science, and put patients, their families, and the community at the centre of everything we do.&nbsp;

POMPE

Our commitment to rare disease 

Nexviadyme®&nbsp;▼(avalglucosidase alfa) prescribing information GB

Myozyme® (alglucosidase alfa) prescribing information GB

From the challenges of cell delivery, to optimising targeting,2,4&nbsp;explore why Sanofi developed Nexviadyme&reg;.

Watch how we used preclinical mice models of Pompe to design Nexviadyme&reg;, and improve maintenance of muscle strength compared to Myozyme&reg;.3,5

Nexviadyme® ▼ (avalglucosidase alfa) prescribing information GB

Myozyme® (alglucosidase alfa) prescribing information GB

Myozyme® (alglucosidase alfa) prescribing information NI

This is intended for HCPs practising in Great Britain (England, Scotland, Wales) only.
This video outlines the development process for Nexviadyme®. Find out how we used preclinical mice models of Pompe to help design Nexviadyme®, and ultimately, improve maintenance of muscle strength compared to Myozyme®.1,2

Watch the below video to find out how we developed Nexviadyme® to ultimately try to improve outcomes for patients with Pompe disease.

How Nexviadyme®▼ (avalglucosidase alfa) was designed

Nexviadyme® (avalglucosidase alfa) prescribing information GB

Find out how we have led with science for over 30 years,3&nbsp;and continue to bring innovation to people living with lysosomal storage disorders.

Watch the below video to find out how we developed Nexviadyme® to ultimately try to improve outcomes for patients with Pompe disease.

Nexviadyme®▼(avalglucosidase alfa) prescribing information GB

Gaucher disease can be classified into three types, which make a phenotypic continuum ranging from mild to severe nervous symptoms.2–4&nbsp;The classic categories of types 1, 2, and 3 have blurred edges along the continuum of the disease.

Adapted from Sidransky E, 2004.&nbsp;Mol Genet Metab.&nbsp;83(1–2):6–15.3

The table summarises aspects of Gaucher disease according to the three different types.1,5

<table>
 <tbody>
 <tr>
 <td style="background-color:#525ca3">&nbsp;</td>
 <td style="background-color:#525ca3">Type 1 Non neuronopathic(≥1/100 to &lt;1/10)</td>
 <td style="background-color:#525ca3">Type 2 Acute neuronopathic (≥1/100 to &lt;1/10)</td>
 <td style="background-color:#525ca3">Type 3 Chronic neuronopathic (≥1/100 to &lt;1/10)</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Prevalence</td>
 <td style="background-color:#e6edf9">1:50,000 – 1:100,000 (pan-ethnic) 
 1:850 (Ashkenazi Jews)</td>
 <td style="background-color:#e6edf9">&lt; 1:150,000 (pan-ethnic)</td>
 <td style="background-color:#e6edf9">&lt; 1:150,000 (pan-ethnic)</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Age at presentation</td>
 <td style="background-color:#e6edf9">Any</td>
 <td style="background-color:#e6edf9">Infancy</td>
 <td style="background-color:#e6edf9">Childhood</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Lifespan</td>
 <td style="background-color:#e6edf9">Variable</td>
 <td style="background-color:#e6edf9">&lt; 2 y</td>
 <td style="background-color:#e6edf9">&lt; 40 y</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Primary CNS disease</td>
 <td style="background-color:#e6edf9">None</td>
 <td style="background-color:#e6edf9">Severe</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Hepatosplenomegaly</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 <td style="background-color:#e6edf9">Severe</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Haematologic 
 abnormalities</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 <td style="background-color:#e6edf9">Severe</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 </tr>
 <tr>
 <td style="background-color:#e6edf9">Osseous symptoms</td>
 <td style="background-color:#e6edf9">Mild to severe</td>
 <td style="background-color:#e6edf9">None</td>
 <td style="background-color:#e6edf9">Mild to moderate</td>
 </tr>
 </tbody>
</table>

Gaucher disease is one of the most common lysosomal storage disorders, affecting an estimated 1 in 40,000 to 1 in 100,000 people around the world.1 It can be diagnosed at any age from infancy to late adulthood. It is an inherited deficiency of the lysosomal enzyme acid-β-glucosidase (glucocerebrosidase, GBA1), which results in the accumulation of glucocerebroside within lysosomes of macrophages.1

What is Gaucher disease?

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<h2>InRange Clinical Summary</h2>
InRange - the first randomised, Phase 4 study to use Time-In-Range (TIR) as the primary endpoint to compare second generation basal insulin analogues Toujeo® and insulin degludec 100U/mL in people with T1DM.

Download this six page clinical summary about the InRange study and learn more about the study design, the key findings and more

TYPE-1-DIABETES

DIABETES

<h2>InRange Study</h2>
Professor Richard Bergenstal presents the results of the InRange study. This study is the first randomised controlled trial to compare second generation basal insulin analogues Toujeo® and insulin degludec 100 U/mL. It compares adults with T1DM, using Time-in-Range (TIR) as the primary endpoint. Watch and learn about the study design and key findings.

InRange Study

<h2>Time in Range Guide</h2>
View the guide below to find out more about the impact of time in range on your patients.&nbsp;

<a href="/uk/type-1-diabetes">SEE MORE FROM SCIENCE</a>

<h3>Toujeo</h3>
Toujeo®&nbsp;is indicated as treatment of diabetes mellitus in adults,&nbsp;adolescents and children from the age of 6 years.&nbsp;

<h3>Patient Website</h3>
Find resources and support for your patients on Toujeo®. Help patients understand the basics of living and staying well with diabetes and how to have a positive start and experience with the medication prescribed to them.

<h2>Get in touch with the Diabetes Team</h2>

Toujeo® (insulin glargine 300 units/mL) Prescribing Information

For over 30 years, Sanofi have worked with patients and doctors to generate data to deliver better care for patients with rare diseases. Watch this video to find out more about the milestones achieved since the establishment of the first rare disease registry in 1991.

<h2>Commitment to Rare Disease Registries</h2>

BETTER-CARE-FOR-RARE

Supporting Real-World Evidence Generation: Rare Disease Registries

<h2>The History of Rare Disease Registries</h2>

Read the publication that highlights the history, process and impact of rare disease registries; the largest observation database for lysosomal storage disorders.

This is intended for HCPs practising in Great Britain (England, Scotland, Wales) only.
In this video, find out how we used glycoengineering and chemical conjugation to help overcome the challenge of cellular delivery of acid alpha-glucosidase, by increasing the levels of bis-M6P1,4&nbsp;compared to Myozyme (alglucosidase alfa).

Why we developed Nexviadyme®▼ (avalglucosidase alfa)

Find out how we have led with science for over 30 years,5&nbsp;and continue to bring innovation to people living with lysosomal storage disorders.

Watch how we used preclinical mice models of Pompe to design Nexviadyme&reg;, and improve maintenance of muscle strength compared to Myozyme&reg;.1,3

 Nexviadyme®▼(avalglucosidase alfa) prescribing information GB

<a href="${link:{uuid:{55f30477-c7e2-49c0-8cfc-16966f28d4c2},repository:{dam},path:{/Portal/UK/Prescribing-links/Praluent-PI-GB-October-2022.pdf}}}" target="_blank">Prescribing Information(GB)</a>

<a href="${link:{uuid:{9e0d6f97-2ebb-4422-92ab-569b89c7dfe0},repository:{dam},path:{/Portal/UK/Prescribing-links/Praluent-PI-NI.pdf}}}" target="_blank">Prescribing Information(NI)</a>

ODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia.
Kastelein JJP, et al. Eur Heart J 2015; 36:2996–3003.
All of the information and data within this section (unless noted) is a summary of this publication.
<ul>
</ul>

<h2>Study objectives</h2>
The ODYSSEY FHI and FH II studies evaluated the long-term efficacy and safety of Praluent®&nbsp;75/150 mg in heterozygous familial hypercholesterolaemia‡ (HeFH) patients with inadequately controlled low-density lipoprotein cholesterol (LDL-C) despite maximally tolerated dose (MTD) statins** ± other lipid-lowering therapies (LLTs).
<ul>
</ul>

<h2>Study design</h2>
ODYSSEY FH I and FH II studies had similar study designs with FH I performed at 89 sites across North America, Europe and South Africa whilst FH II was performed across 26 sites in Europe.
Both FH I and FH II were 78 week, multicentre, double-blind, placebo-controlled, parallel group, phase III studies involving 735 adult HeFH‡ patients (FH I, n=486; FH II, n=249) who were receiving MTD statins** ± other LLTs with:
<ul>
 <li>LDL-C ≥1.8 mmol/L in patients with prior cardiovascular events†</li>
</ul>
or
<ul>
 <li>LDL-C ≥2.6 mmol/L in patients without a history of CV events</li>
</ul>
Patients were randomised to subcutaneous Praluent®&nbsp;75 mg every 2 weeks (Q2W) (FH I, n=323; FH II, n=167) or placebo Q2W (FH I, n=163; FH II, n=82) (Figure 1). Praluent®&nbsp;dose was increased to 150 mg Q2W at Week 12 if Week 8 LDL-C was ≥1.8 mmol/L.
The primary efficacy endpoint was the percentage change in LDL-C from baseline to Week 24. Secondary endpoints included:
<ul>
 <li>Proportion patients achieving the pre-specified LDL-C level target of &lt;1.8 mmol/L at Week 24</li>
 <li>Percentage change in LDL-C from baseline to Week 78</li>
 <li>Safety - including incidence of treatment-emergent adverse events (TEAEs), treatment-emergent serious AEs (SAEs), injection site reactions and adjudicated CV events.</li>
</ul>

Figure 1: ODYSSEY FHI and FH II: Study design&nbsp;- Adapted from Kastelein JJP, et al. Eur Heart J 2015; 36:2996–30031

<h3>Baseline characteristics</h3>
Baseline characteristics were well balanced between the treatment groups (Table 1). In FH I and FH II, 85.8% and 91.6% of Praluent patients received study treatment for ≥76 weeks versus 87.7% and 90.1% of placebo.

<table cellpadding="20" style="width:100%;">
 <tbody>
 <tr>
 <td colspan="1" rowspan="2" style="background-color: rgb(0, 155, 195); width: 40%;">
 &nbsp;
 &nbsp;</td>
 <td style="background-color:#009bc3">
 Praluent 
 75/150 mg 
 (n=323)</td>
 <td style="background-color:#009bc3">
 Placebo 
 (n=163)</td>
 <td style="background-color:#009bc3">
 Praluent 
 75/150 mg 
 (n=167)</td>
 <td style="background-color:#009bc3">Placebo 
 (n=82)</td>
 </tr>
 <tr>
 <td colspan="2" rowspan="1" style="background-color:#009bc3">
 FH I</td>
 <td colspan="2" rowspan="1" style="background-color:#009bc3">
 FH II</td>
 </tr>
 <tr>
 <td colspan="5">
 Demographics</td>
 </tr>
 <tr>
 <td>
 Age in years, mean ±SD</td>
 <td>
 52.1 ± 12.9</td>
 <td>
 51.7 ± 12.3</td>
 <td>
 53.2 ± 12.9</td>
 <td>
 53.2 ± 12.5</td>
 </tr>
 <tr>
 <td>
 Male, %</td>
 <td>
 55.7</td>
 <td>
 57.7</td>
 <td>
 51.5</td>
 <td>
 54.9</td>
 </tr>
 <tr>
 <td colspan="5">
 CV history/risk factors</td>
 </tr>
 <tr>
 <td>
 CHD history*, %</td>
 <td>
 45.5</td>
 <td>
 47.9</td>
 <td>
 34.7</td>
 <td>
 37.8</td>
 </tr>
 <tr>
 <td>
 CHD risk&nbsp;equivalent†, %</td>
 <td>
 16.7</td>
 <td>
 15.3</td>
 <td>
 9.0</td>
 <td>
 4.9</td>
 </tr>
 <tr>
 <td>
 Type 2 diabetes, %</td>
 <td>
 9.9</td>
 <td>
 15.3</td>
 <td>
 4.2</td>
 <td>
 3.7</td>
 </tr>
 <tr>
 <td colspan="5">
 Lipid medication</td>
 </tr>
 <tr>
 <td>
 Any statin, %</td>
 <td>
 100</td>
 <td>
 100</td>
 <td>
 100</td>
 <td>
 100</td>
 </tr>
 <tr>
 <td>
 High dose statin§, %</td>
 <td>
 82.7</td>
 <td>
 85.3</td>
 <td>
 86.8</td>
 <td>
 91.5</td>
 </tr>
 <tr>
 <td>
 Ezetimibe, %</td>
 <td>
 56.0</td>
 <td>
 59.5</td>
 <td>
 67.1</td>
 <td>
 64.6</td>
 </tr>
 <tr>
 <td colspan="5">
 Lipid parameters</td>
 </tr>
 <tr>
 <td>
 LDL-C, (calculated) mmol/L ± SD</td>
 <td>
 3.7 ± 0.1</td>
 <td>
 3.7 ± 0.1</td>
 <td>
 3.5 ± 0.1</td>
 <td>
 3.5 ± 0.1</td>
 </tr>
 </tbody>
</table>

HD = coronary heart disease; CV = cardiovascular; LLT = lipid-lowering therapy, SD= standard deviation. *CHD history defined as acute myocardial infarction, silent myocardial infarction, unstable angina, or coronary revascularization. †CHD risk equivalent; Ischaemic stroke, peripheral arterial disease, moderate chronic kidney disease (estimated glomerular filtration rate of ≥30 and ≤60 mL/min/1.73 m2), and diabetes with two or more risk factors. §High-dose statin therapy defined as daily doses of 40–80 mg atorvastatin, 20–40 mg rosuvastatin or 80 mg simvastatin.

<h3>Key efficacy results</h3>
In adults with HeFH, Praluent75/150 mg 2QW led to significantly greater reductions in LDL-C compared to placebo (Figure 2), allowed a significantly greater proportion of patients to achieve a pre-specified LDL-C target compared to placebo (Figure 3) and LDL-C reductions were consistent and sustained over 78 weeks (Figure 4):
<ul>
 <li>At Week 24, mean percentage change in LDL-C was -48.8% (1.9mmol/L) (FH I) and -48.7% (1.74mmol/L) (FH II) for Praluent versus 9.1% (0.29mmol/L) (FH I) and 2.8% (0.07mmol/L) (FH II) for placebo (p&lt;0.0001 for both studies) (Figure 2)</li>
 <li>At Week 24, regardless of prior CV status, 59.8% (FH I) and 68.2% (FH II) of Praluent patients achieved pre-specified LDL-C target of &lt;1.8 mmol/L versus 0.8% (FH I) and 1.2% (FH II) of placebo patients (p&lt;0.0001 for both studies) (Figure 3)</li>
 <li>At Week 78, the mean reduction in LDL-C level with Praluent compared with placebo was -51.8% (1.9mmol/L) (FH I)&nbsp; and -52.1% (1.76 mmol/L) (FH II) (Figure 4).</li>
</ul>
Amongst Praluent patients who received double-blind treatment for at least 12 weeks, 56.6% (FH I) and 61.4% (FH II) had LDL-C levels &lt;1.8 mmol/L at Week 8 and therefore remained on Praluent 75 mg Q2W for the duration of the study. LDL-C responses were durable and were maintained (up to 78 weeks) in Praluent treated patients.

Figure 2: ODYSSEY FH I and FH II: Mean reduction in LDL-C from baseline to Week 52&nbsp;- Adapted from Kastelein JJP, et al. Eur Heart J 2015; 36:2996–30031

Placebo + MTD statin therapy ± other LLT 
 Praluent + MTD statin therapy ± other LLT *Praluent dose was 75 mg administered SC every two weeks, but if LDL-C level was ≥1.8 mmol/L at Week 8, a pre-specified dose up-titration to 150 mg every two weeks was performed at Week 12 †This number refers to the number of patients receiving Praluent (ITT analysis)1 
 ‡This number refers to the number of patients receiving placebo (ITT analysis)1

Figure 3: ODYSSEY FH I and FH II: Proportion of patients achieving LDL-C target at Week 24&nbsp;- (Adapted from Kastelein JJP, et al. Eur Heart J 2015; 36:2996–30031)

Placebo + MTD statin therapy ± other LLT 
 Praluent + MTD statin therapy ± other LLT *Praluent dose was 75 mg administered SC every two weeks, but if LDL-C level was ≥1.8 mmol/L at Week 8, a pre-specified dose up-titration to 150 mg every two weeks was performed at Week 12 †This number refers to the number of patients receiving Praluent (ITT analysis)1 
 ‡Intention to treat (ITT) analysis 
 &nbsp;

Figure 4: ODYSSEY FH I and FH II: Mean reduction in LDL-C from baseline to Week 781,2&nbsp;- (Adapted from Kastelein JJP, et al. Eur Heart J 2015;36:2996-3003)

Placebo + MTD statin therapy ± other LLT 
 Praluent + MTD statin therapy ± other LLT ‡Intention to treat (ITT) analysis
 
 *Praluent dose was 75 mg administered SC every two weeks, but if LDL-C level was ≥ 1.8 mmol/L at Week 8, a pre-specified dose up-titration to 150 mg every two weeks was performed at Week 121 
 †Statin therapy at the maximum-tolerated dose (40-80 mg atorvastatin, 20-40 mg rosuvastatin daily or 80 mg simvastatin [if on this dose for &gt;1 year]) or a lower dose, provided a reason was documented

<h2>Key safety results</h2>
Frequency of TEAEs and SAEs were similar in Praluent and placebo patients (Table 2). Although a higher proportion of Praluent than placebo patients experienced injection site reactions, these differences were non-significant (FH I, p=0.77; FH II, p=0.38); most reactions were mild and none lead to discontinuation (Table 2).

Table 2: ODYSSEY FH I and FH II: Treatment-emergent adverse events (safety population)&nbsp;- Adapted from Kastelein JJP, et al. Eur Heart J 2015; 36:2996–30031

<table cellpadding="20" style="width:100%;">
 <tbody>
 <tr>
 <td colspan="1" rowspan="2" style="background-color: rgb(0, 155, 195); width: 40%;">
 &nbsp;
 &nbsp;</td>
 <td style="background-color: rgb(0, 155, 195); width: 10%;">
 Praluent 
 75/150 mg 
 (n=322)</td>
 <td style="background-color: rgb(0, 155, 195); width: 10%;">
 Placebo 
 (n=163)</td>
 <td style="background-color: rgb(0, 155, 195); width: 10%;">
 Praluent 
 75/150 mg 
 (n=167)</td>
 <td style="background-color: rgb(0, 155, 195); width: 10%;">Placebo 
 (n=81)</td>
 </tr>
 <tr>
 <td style="background-color:#009bc3">
 FH I</td>
 <td style="background-color:#009bc3">
 &nbsp;</td>
 <td style="background-color:#009bc3">
 FH II</td>
 <td style="background-color:#009bc3">
 &nbsp;</td>
 </tr>
 <tr>
 <td>
 TEAE, n (%)</td>
 <td>
 263 (81.7)</td>
 <td>
 129 (79.1)</td>
 <td>
 125 (74.9)</td>
 <td>
 66 (81.5)</td>
 </tr>
 <tr>
 <td>
 Treatment-emergent SAEs, n (%)</td>
 <td>
 44 (13.7)</td>
 <td>
 22 (13.5)</td>
 <td>
 15 (9.0)</td>
 <td>
 8 (9.9)</td>
 </tr>
 <tr>
 <td>
 TEAE leading to discontinuation, n (%)</td>
 <td>
 11 (3.4)</td>
 <td>
 10 (6.1)</td>
 <td>
 6 (3.6)</td>
 <td>
 1 (1.2)</td>
 </tr>
 <tr>
 <td>
 Injection site reactions, n (%)</td>
 <td>
 40 (12.4)</td>
 <td>
 18 (11.0)</td>
 <td>
 19 (11.4)</td>
 <td>
 6 (7.4)</td>
 </tr>
 <tr>
 <td>
 TEAEs leading to death, n (%)</td>
 <td>
 6 (1.9)</td>
 <td>
 0</td>
 <td>
 0</td>
 <td>
 0</td>
 </tr>
 <tr>
 <td colspan="5">
 Safety events of interest</td>
 </tr>
 <tr>
 <td>
 Positively adjudicated CV adverse events, n (%)</td>
 <td>
 8 (2.5)</td>
 <td>
 3 (1.8)</td>
 <td>
 2 (1.2)</td>
 <td>
 1 (1.2)</td>
 </tr>
 <tr>
 <td>
 General allergic TEAEs, n (%)</td>
 <td>
 28 (8.7)</td>
 <td>
 16 (9.8)</td>
 <td>
 19 (11.4)</td>
 <td>
 5 (6.2)</td>
 </tr>
 <tr>
 <td>
 Neurological TEAEs, n (%)</td>
 <td>
 12 (3.7)</td>
 <td>
 7 (4.3)</td>
 <td>
 7 (4.2)</td>
 <td>
 2 (2.5)</td>
 </tr>
 <tr>
 <td>
 Neurocognitive disorders, n (%)</td>
 <td>
 2 (0.6)</td>
 <td>
 2 (1.2)</td>
 <td>
 0</td>
 <td>
 1 (1.2)</td>
 </tr>
 <tr>
 <td>
 Development/ 
 worsening of diabetes, n (%)</td>
 <td>
 6 (1.9)</td>
 <td>
 4 (2.5)</td>
 <td>
 4 (2.4)</td>
 <td>
 2 (2.5)</td>
 </tr>
 <tr>
 <td>
 Ophthalmologic disorders, n (%)</td>
 <td>
 3 (0.9)</td>
 <td>
 4 (2.5)</td>
 <td>
 3 (1.8)</td>
 <td>
 1 (1.2)</td>
 </tr>
 <tr>
 <td>
 Alanine aminotransferase, &gt;3 x ULN (%)</td>
 <td>
 5/322 (1.6)</td>
 <td>
 2/163 (1.2)</td>
 <td>
 6/166 (3.6)</td>
 <td>
 1/81 (1.2)</td>
 </tr>
 <tr>
 <td>
 Creatine kinase, &gt;3 x ULN (%)</td>
 <td>
 13/318 (4.1)</td>
 <td>
 10/163 (6.1)</td>
 <td>
 8/165 (4.8)</td>
 <td>
 6/80 (7.5)</td>
 </tr>
 </tbody>
</table>

Please refer to publication for full safety profile information
CV = cardiovascular; SAEs, serious adverse events; TEAE = treatment-emergent adverse events

<h2>Conclusions</h2>
<ul>
 <li>Praluent 75/150 mg every 2 weeks significantly lowered calculated LDL-C from baseline to Week 24 in patients with HeFH on top of the maximum-tolerated dose of statin compared to placebo (-48.8% (1.9mmol/L) (FH I) and -48.7% (1.74mmol/L) (FH II) for Praluent versus 9.1% (0.29mmol/L) (FH I) and 2.8% (0.07mmol/L) (FH II) p &lt;0.0001 for both studies)</li>
 <li>At Week 24, 59.8% of patients in the FHI study and 68.2% in the FHII study achieved the pre-specified target LDL-C level of &lt;1.8 mmol/L compared to 0.8% (FHI) and 1.2% (FHII) patients on placebo (p&lt;0.0001 for both studies)</li>
 <li>At week 8, over 50% of patients achieved pre-specified LDL-C target of &lt;1.8 mmol/L and were maintained on the 75mg dose</li>
 <li>The incidences of TEAEs and treatment-emergent SAEs were similar between treatment groups over 78 weeks of treatment 
 &nbsp;</li>
</ul>
‡HeFH diagnosed by either genotyping or clinical criteria (Simon Broome or World Health Organization/Dutch Lipid clinic network score of &gt;8)
**Maximum-tolerated statin dose defined as 40–80 mg atorvastatin, 20–40 mg rosuvastatin or 80 mg simvastatin, or lower dose if documented reason provided e.g., intolerance or local practice
†Established CHD defined as acute myocardial infarction, silent myocardial infarction, unstable angina, coronary revascularisation, or clinically significant CHD diagnosed by invasive or non-invasive testing. CHD risk equivalents defined as ischaemic stroke, peripheral artery disease, moderate chronic kidney disease, or diabetes (only if ≥2 risk factors present)
§High-dose statin therapy defined as 40–80 mg atorvastatin, 20–40 mg rosuvastatin or 80 mg simvastatin

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