What is

AELMHU?

We are a non-profit association made up of pharmaceutical companies specializing in rare diseases that are firmly committed to discovering, researching, developing, and marketing innovative therapies that improve the lives of patients and their families.

Access reports

At AELMHU, we produce quarterly and annual reports on access to orphan drugs in Europe and Spain (
).

Check here for our latest update

NEWS

January 14, 2026

AELMHU celebrates 15 years of commitment to people with rare diseases

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) celebrates its 15th anniversary today, a milestone that reaffirms the commitment of pharmaceutical companies dedicated to researching and developing treatments for ... Read more

IV Jornada sobre Terapias Avanzadas para Enfermedades Raras

AELMHU en la constitución de la Alianza de la Industria Farmacéutica

Jornada Multidisciplinar de Enfermedades Raras en el Hospital Gregorio Marañón

AELMHU participates in the FARPE meeting at CREER

The application period for the 8th edition of the AELMHU Awards is now open.

Report: ‘The social value of orphan drugs’

INTERVIEWS

Déficit de Alfa-1: la importancia de diagnosticar antes y mejor

24/04/2026

Fibrosis quística: una nueva realidad en la infancia y en la edad adulta

22/04/2026

Inmunodeficiencias primarias: 550 enfermedades raras del sistema inmune

21/04/2026

La hemofilia y otras coagulopatías en mujeres: más allá de ser “portadoras”

17/04/2026

Enfermedad de Pompe: visibilidad para avanzar en diagnóstico y acceso

15/04/2026

“Cushing, the Invisible Thief”: The Story of Florina Tudorache

April 8, 2026

Fabry disease: unmet needs among patients

April 6, 2026

Lipodystrophies: From Underdiagnosis to a Multidisciplinary Approach

March 31, 2026

Women in the rare disease pharmaceutical industry: talent and leadership

March 6, 2026

‘Because every person matters’: equity, key in rare diseases

February 27, 2026

ASEM Federation: challenges and advances in neuromuscular diseases

14/11/2025

NET Spain: a decade of giving voice to patients with neuroendocrine tumors

11/11/2025

Familial chylomicronemia: challenges and approach to this rare disease

07/11/2025

Empathy and accompaniment: the keys to DEBRA Piel de Mariposa's work

27/10/2025

Hope in the face of HPN: the story and life of Adriana Reyes

13/10/2025

Progressive familial intrahepatic cholestasis: What is it and what are its causes?

06/10/2025

Homozygous familial hypercholesterolemia: why early diagnosis is key

24/09/2025

"Strength in numbers": the importance of patients in rare diseases

19/09/2025

When science and patients come together: achievements and remaining challenges in cystic fibrosis.

08/09/2025

What is Familial Mediterranean Fever

04/08/2025

Living with sickle cell disease

19/06/2025

Interview with Raquel Pardo, president of AMES

June 2, 2025

World Neurofibromatosis Day: the invisible challenge patients face

16/05/2025

Chema: living with MPS1 and never giving up

14/05/2025

World Nursing Day: the link that transforms hospital care

12/05/2025

Thalassemia major: symptoms, diagnosis and treatment through a real-life testimonial

May 8, 2025

Interview with Enrique Carazo: visibility in the face of pulmonary hypertension

05/05/2025

Fabry disease: the clinical voice

30/04/2025

The importance of diagnosis

29/04/2025

What is Fibrodysplasia Ossificans Progressiva?

23/04/2025

What is Hemophilia

April 16, 2025

Living with Pompe disease

April 15, 2025

Living with Fabry

April 8, 2025

What are lipodystrophies?

March 31, 2025

pERsonas, theme for World Rare Disease Day 2025

20/02/2025

What is Alagille Syndrome

21/01/2025

The importance of mental health in rare disease patients

13/01/2025

World Doctors' Day

03/12/2024

What is Alpha Mannosidosis

22/11/2024

What is it like to live with X-linked Hypophosphatemia?

23/10/2024

Life with Familial Chylomicroenemia Syndrome

31/10/2024

Living with Acid Sphingomyelinase Deficiency (ASMD)

18/10/2024

Interview with Begoña Polo, expert in Progressive Familial Intrahepatic Intrahepatic Cholestasis

04/10/2024

Living with Gaucher disease

30/09/2024

Immune thrombocytopenia: an invisible challenge

30/09/2024

Living with PTTa

20/09/2024

An informed patient is half cured

12/09/2024

Life with primary biliary cholangitis

09/09/2024

ASSOCIATED COMPANIES

RESEARCH AND COMMITMENT to rare diseases

MEMBERS

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What is an orphan drug?

Orphan drugs (ODs) are innovative treatments, many of them biotechnological in origin, intended to diagnose, prevent, or treat patients with rare diseases for which there is no alternative therapy.