The Spanish Association of Orphan and Ultra Orphan Drug Laboratories(AELMHU) has presented the VII edition of its annual awards, with which it recognizes the most outstanding contributions of institutions, patient associations and health professionals in the field of rare diseases.

Doctors María Luz Couce Pico, Pascual Sanz Bigorra and Patricia Smeyers Durá, the Andalusian Association of Hemophilia(ASANHEMO) and the Association of People with Achondroplasia and other Skeletal Dysplasias in Spain(ADEE) received the awards in this seventh edition.

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The Spanish Association of Orphan and Ultra Orphan Drug Laboratories(AELMHU) has announced this Thursday the decision of the Jury of the VII edition of the AELMHU Awards, which aim to recognize the contribution of institutions, patient associations and health professionals in the field of rare diseases.

Dr. María Luz Couce Pico received the AELMHU 2025 honorary award, while Dr. Pascual Sanz Bigorra and Dr. Patricia Smeyers Durá were recognized, respectively, for their research and communication, dissemination and/or awareness-raising work on rare diseases.

The Andalusian Hemophilia Association, for its "Comprehensive care and rehabilitation program for people with hemophilia and congenital coagulopathies", and the Association of People with Achondroplasia and other Skeletal Dysplasias in Spain (ADEE), with "Los Bufones de Velázquez", complete the awards of this seventh edition.

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The National Association of Health Informers (ANIS) and the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU), the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) have organized this Wednesday the II Training Day on rare diseases, aimed at journalists specialized in health.

At the event, AELMHU's vice president, María José Sánchez, presented to journalists the data from the first 2025 Orphan Drug Access Quarterly Report, which analyzes the access situation for trade-name products with current orphan designation as of April 30, 2025.

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The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has published a new report that analyzes the most relevant data on clinical trials authorized for rare pathologies and their evolution over the last five years in our country. The launch is framed in the context of the International Clinical Trials Day.

According to the data, a total of 929 clinical trials were authorized in Spain in 2024. Of these, 207 trials were aimed at rare diseases. A comparison of the number of clinical trials for rare diseases authorized in 2024 with that of 2023 shows an increase of 10% in the latter year. This increase is good news for the country, and especially for patients who are awaiting the arrival of a treatment, in many cases the first to treat their disease.

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The Spanish Association of Orphan and Ultra Orphan Drug Laboratories(AELMHU) has presented the VII edition of its awards, created with the aim of promoting knowledge about rare diseases and orphan drugs. These awards annually highlight the work of experts and organizations by recognizing their professional careers and the most outstanding projects in assistance, dissemination and awareness of these pathologies.

In previous editions, AELMHU has recognized the work of such relevant personalities in the world of rare pathologies as Ángel Carracedo, Francesc Palau, Julio Sánchez-Fierro, Josep Torrent-Farnell, Magdalena Ugarte, and José Luis Poveda, among others.

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The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) held today the III National Conference: The Future of Advanced Therapies for Rare Diseases, with the aim of promoting dialogue and critical reflection among the different agents of the healthcare system in order to highlight the development of advanced therapies for rare diseases and access for patients in Spain. The conference was made possible thanks to the collaboration of: Amgen, CSL Behring, PTC Therapeutics, Ultragenyx Pharmaceutical and Vertex.

Among the main conclusions, the participants agreed on the importance of the therapeutic and social value of advanced therapies and the need to work together to improve the approach to rare diseases. Facing the great challenges, both at European and national or regional level, concerning diagnosis, research, development, financing and access to these treatments.

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) and the General Council of Pharmaceutical Associations (CGCOF) have signed a collaboration agreement to promote the skills of pharmacists in this type of drugs for the prevention, diagnosis or treatment of rare diseases.

The president of the General Council of Pharmaceutical Associations, Jesús Aguilar, and the president of AELMHU, Beatriz Perales, have signed the document that includes the joint organization of training or informative activities, as well as health programs and campaigns. The first action to be promoted by both entities is the launch of the "Pharmacy and Orphan Drugs" channel, within the video advice channel #TuFarmacéuticoInforma that the General Council of Pharmaceutical Associations has on YouTube.

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The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories(AELMHU) today presents the results of its Annual Report on Access to Orphan Drugs for 2024, which every year provides information on the situation of these drugs in Europe and their availability for patients in Spain.

The report analyzes the data on orphan drugs based on different European and Spanish public sources as of December 31, 2024, taking into account the evolution of the indicators.

According to the analysis, the situation in Europe has shown a positive evolution, with 25 new drugs with orphan designation and trade name, 6 more than in 2023. In addition, 17 drugs obtained marketing authorization in the European Union from the European Medicines Agency (EMA), improving the number of authorized products by 5 points over the previous year.

The Spanish Association of Orphan and Ultra Orphan Drug Laboratories(AELMHU) has presented its annual awards, with which it recognizes the most outstanding contributions of institutions, patient associations and health professionals to the field of rare diseases in order to improve the health and quality of life of millions of people in our country.

Inaugurated by the president of AELMHU, Beatriz Perales, and conducted by the journalist María Rey, the award ceremony was held in Madrid in the presence of representatives from the institutional sphere, the industry, patients and the scientific and healthcare community. It was closed by César Hernández, Director General of the Common Portfolio of Services of the National Health System and Pharmacy of the Ministry of Health. The event included numerous expressions of remembrance and affection for all those affected by the recent DANA.

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The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) has announced today the decision of the Jury of the VI Edition of its Awards, made up of thirteen professionals of recognized prestige in the field of rare diseases.

With these awards, AELMHU recognizes the contribution of institutions, patient associations and health professionals in the field of rare pathologies, in order to continue recognizing year after year the most relevant contributions both in the visibility and awareness of these pathologies and in their research and treatments.

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