<h3>Sanofi’s vision for Better Care for Rare: a world where diagnosis, treatment and support for rare diseases is equitable and accessible, where the chance to live well with a rare disease has no exception, no matter who people are or where they are in their journey.</h3>
We, at Sanofi, hope to fulfil this vision through close alignment with the England Rare Diseases Action Plans in order to help solve the&nbsp;complex and unique challenges inherent in rare diseases.&nbsp;
Hear from experts on the future of rare diseases, including their thoughts and opinions on how clinical practice&nbsp;and services need to change to fulfil the priorities set out in the Rare Disease Framework.&nbsp;

Specialist Pharmacist in Inherited Metabolic Disease

Consultant in Inherited Metabolic Diseases at GOSH

Consultant in Metabolic Medicine at Salford Royal Hospital

BETTER-CARE-FOR-RARE

RARE-DISEASE

Fulfilling priorities in England Rare Disease Framework. How can we make them a reality?

<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

GAUCHER

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

<a href="${link:{uuid:{c90870f5-b8bd-4138-bee3-88766d4cee04},repository:{dam},path:{/Portal/UK/Prescribing-links/Clexane-pi.pdf}}}" target="_blank">Prescribing Information UK</a>

Clexane is indicated in adults for the extended treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE) and prevention of its recurrence in patients with active cancer based on the RIETECAT trial results.
RIETECAT trial is a multinational observational retrospective cohort study using data from the RIETE registry.1
It is the first and largest&nbsp;study comparing the effectiveness and safety of enoxaparin against dalterparin and tinzaparin in a real-world cohort of cancer patients with VTE.1

<h2>RIETECAT outcomes</h2>
Cancer patients with VTE receiving full dose enoxaparin or tinzaparin/dalteparin had comparable effectiveness and safety outcomes over a 6 month period.
OR: 0.79 (95%CI. 049-1.28) p=0.004

The efficacy outcome was a composite of symptomatic DVT and fatal or non-fatal PE and the safety outcome was a composite of major bleeding events (fatal or non fatal) and non major bleeds of clinical significance.
Due to the non-interventional nature of the study, comparability of patients between groups may be difficult to achieve; however multivariate Cox model was used to minimize the confounding bias.
Patients treated with enoxaparin had a non-significantly lower risk for VTE recurrences, a non-significantly higher risk for major bleeding, and similar risks for non-major bleeding or death over 6 months compared to tinzaparin/dalteparin.

<h2>Footnote</h2>
CAT=cancer-associated thrombosis;&nbsp;VTE=Venous Thromboembolism;&nbsp;CI=confidence interval;&nbsp;OR=Odds Ratio;&nbsp;SC=subcutaneous;&nbsp;IU=international unit;&nbsp;OD=Once daily;&nbsp;PE&nbsp;pulmonary embolism;&nbsp;NMCR=Non-major clinically relevant.

<a href="/uk/thrombosis">SEE MORE FROM SCIENCE</a>

Get in touch with the Thrombosis Team

Clexane® (enoxaparin) Prescribing Information UK

THROMBOSIS

CARDIOLOGY

Treatment of CAT Clexane (enoxaparin)


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?

Gaucher Talking Heads Video

Gaucher Talking Heads Video - Professor Tim Cox

&nbsp;Professor Tim Cox&nbsp;–&nbsp;Consultant Physician, Addenbrooke’s Hospital, Cambridge

Professor Tim Cox provides a guide to Gaucher Disease and the importance of testing for Haematologists

<a href="${link:{uuid:{8cd02cf2-bd72-495f-b720-698ef8d3f0da},repository:{dam},path:{/Portal/UK/Prescribing-links/Toujeo.pdf}}}" target="_blank">Prescribing Information Toujeo (insulin glargine 300 units/mL)</a>

Ask us your questions about our products, patient materials or organising a meeting.

Sanofi is here to support you and your patients.

<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>

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.

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Development of ADCT involved literature review as well as interviews with patients and physicians, and was funded by Sanofi and Regeneron.

The ADCT gives a measure of how controlled your patient’s eczema is. Use these 6 concise questions to evaluate all dimensions of atopic dermatitis control.

Atopic dermatitis, a type of eczema, may be affecting your patient’s life in more ways than you know.

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