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.

ㅤㅤInformes deㅤ ㅤacceso

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

Check here for our latest update

Informes sobre ensayos clínicos

AELMHU publica su Informe Anual sobre Ensayos Clínicos para Enfermedades Raras, donde se analizan sobre los ensayos autorizados para estas patologías.

Check here for our latest update

Informe valor social de los MM.HH.

Informe “Aspectos diferenciales de los medicamentos huérfanos y su valor desde una perspectiva social” elaborado desde la Fundación Weber con el apoyo de AELMHU.

Check here for our latest update

NEWS

January 14, 2026

AELMHU celebrates 15 years of commitment to people with rare diseases

La Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos (AELMHU) cumple 15 años, un hito que reafirma el compromiso de las compañías farmacéuticas dedicadas a investigar y desarrollar tratamientos para las personas con enfermedades … Leer más

AELMHU hace público el fallo del Jurado de la VIII edición de los Premios AELMHU

AELMHU y otras seis organizaciones de medicamentos y cosmética respaldan revisar la Directiva de Aguas Residuales

AELMHU participa en el XI Congreso Internacional de Medicamentos Huérfanos y Enfermedades Raras de FEDER

AELMHU calls for orphan and ultra-orphan drugs to be excluded from the sixth additional provision

El sector farmacéutico de enfermedades raras prevé un aumento del empleo en España en 2026

The media: Key partners in raising awareness of rare diseases

INTERVIEWS

Investigar, acompañar y sensibilizar: los retos pendientes en Duchenne

07/09/2026

Enfermedades autoinflamatorias: 80 patologías con retos comunes

27/08/2026

Dr. Mª Luz Couce: “Newborn screening saves lives. We need to keep improving it.”

June 26, 2026

“Donating blood saves lives”: the reality for people with thalassemia

June 12, 2026

The Importance of Patient Experts in Rare Diseases

May 14, 2026

616874, the code for people with undiagnosed rare diseases

April 29, 2026

Alpha-1 Deficiency: The Importance of Earlier and More Accurate Diagnosis

April 24, 2026

Cystic Fibrosis: A New Reality in Childhood and Adulthood

April 22, 2026

Primary immunodeficiencies: 550 rare diseases of the immune system

April 21, 2026

Hemophilia and Other Coagulation Disorders in Women: Beyond Being “Carriers”

April 17, 2026

Pompe Disease: Raising Awareness to Improve Diagnosis and Access

April 15, 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

Porphyria: The Emotional and Social Impact of Rare Diseases

May 18, 2026

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.