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Rare Disease Day 2024: New video aims to raise awareness of …

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Rare Disease Day 2024: New video aims to raise awareness of …

29.02.2024 – 10:31


Barcelona/Langenfeld (Rhineland) (ots)

To create greater understanding and awareness of rare diseases such as leukodystrophy, including x-linked adrenoleukodystrophy (X-ALD) and in particular cerebral adrenoleukodystrophy (cALD), the two companies Neuraxpharm and Minoryx have released a new information film. Neuraxpharm is a leading European pharmaceutical company focused on the treatment of central nervous system (CNS) diseases and Minoryx is a biotech company focused on the clinical development of novel therapies for rare CNS diseases with high unmet medical need. On Rare Disease Day on February 29, 2024, both reaffirm their commitment to finding better treatment options for patients suffering from rare diseases such as X-ALD and cALD. The new film can be found on the Neuraxpharm and Minoryx websites as well as on the YouTube and LinkedIn channels (link for example:

Professor Fanny Mochel, a world-leading expert in the field of adrenoleukodystrophy, worked on the film. She is director of the reference center for leukodystrophies at La Pitié-Salpêtrière hospital in Paris. In the film she describes the disease and the need for regular screening for early identification. In addition, Karen Harrison, Support Services Manager of “Alex – The Leukodystrophy Charity” (Alex-TLC), speaks impressively in the film about the experiences of patients and the need for better treatment options.

“The impact of this disease on patients and their families cannot be overstated,” said Dr. Jörg-Thomas Dierks, CEO of Neuraxpharm. “At Neuraxpharm, we are committed to providing better treatment so we can improve the lives of those affected by this devastating disease. We hope this film increases awareness and understanding of the disease.”

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Marc Martinell, CEO of Minoryx, stated: “The X-ALD community deserves our full support to help them deal with the extremely difficult consequences of this disease, which in the case of cALD can also be fatal. We want to raise awareness the diseases and the importance of regular monitoring and early detection, as these are crucial factors for the timely treatment of these patients.”

About X-ALD and cALD

X-ALD is a rare neurodegenerative disease with a global incidence of approximately 6-8/100,000 live births. As a disease linked to the X chromosome, it occurs predominantly in men. However, women are also affected by the chronic form, even if they only develop symptoms later in life. Boys and adult men with X-ALD can develop cALD at any time in their lives, which is characterized by demyelinating brain lesions that progress rapidly and can lead to acute neurological decline and death. These lesions can cause serious symptoms such as loss of voluntary movements, inability to swallow, loss of communication, cortical blindness and total incontinence, and death, with an average survival time of 3 to 4 years.

Progressive cALD occurs in 31-35 percent of childhood ALD patients and typically begins between ages 2 and 12; Up to 60 percent of adult patients with X-ALD develop progressive cALD over time. There is currently no pharmacological treatment for cALD. In childhood, hematopoietic stem cell transplantation (HSCT) can stop the disease. However, this is an aggressive procedure that is only available for some patients. Autologous HSCT is not available worldwide; it still requires hemoablation therapy with associated comorbidities and long-term safety data are still being collected. In adults, experience with HSCT is very limited and the procedure is often not recommended.

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In addition, all X-ALD patients who reach adulthood develop adrenomyeloneuropathy (AMN), which is characterized by progressive spastic paraparesis as well as progressive deterioration of balance and sensory function and the development of incontinence. This form is chronic, symptoms typically appear in adulthood, affects both men and women and has a poor prognosis.

About Neuraxpharm – the European CNS specialist

Neuraxpharm is a leading European specialty pharmaceutical company specializing in the treatment of the central nervous system (CNS), including psychiatric and neurological diseases. The company has a unique understanding of the CNS market, built over 35 years.

Neuraxpharm is constantly seeking new products and solutions for unmet patient needs and expanding its portfolio through its pipeline, partnerships and acquisitions.

The company employs approximately 1,000 people and develops and sells CNS products through a direct presence in more than 20 countries in Europe, two in Latin America and worldwide through partners in more than 40 countries. Neuraxpharm is supported by funds advised by Permira.

Neuraxpharm manufactures many of its pharmaceutical products at Neuraxpharm Pharmaceuticals (formerly Laboratorios Lesvi) in Spain.

For more information please visit www.neuraxpharm.com.

About Minoryx

Minoryx is a biotech company focused on the clinical development of novel therapies for rare CNS diseases with high unmet medical needs. The company’s leading development program includes compounds for X-linked adrenoleukodystrophy (X-ALD) and other rare CNS diseases. The company is backed by a consortium of experienced investors including Columbus Venture Partners, CDTI Innvierte, Caixa Capital Risc, Fund+, Ysios Capital, Roche Venture Fund, Kurma Partners, Chiesi Ventures, SRIW, Idinvest Partners / Eurazeo, SFPI-FPIM, HealthEquity , Sambrinvest and Herrecha, and is supported by a network of other organizations.

Minoryx was founded in 2011 with headquarters in Spain and locations in Belgium and has so far raised more than 120 million euros in financing rounds.

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For more information, visit www.minoryx.com.

Press contact:

Pares Nuri, medical relations GmbH
Hans-Böckler-Straße 46
D-40764 Langenfeld
Tel: +49 2173 9769 22
[email protected]

Optimum Strategic Communications
Nick Bastin / Charlotte Hepburne-Scott / Elena Bates
Tel: +44 (0)203 882 9621
[email protected]

Original content from: Neuraxpharm, transmitted by news aktuell

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