Resources

The Medicine Access Masterclass is a unique overview of early medicine access options of innovative medicine which cannot be found anywhere else.

Paxlovid is an antiviral administered orally to patients who are ill with COVID-19 or have been exposed to the coronavirus SARS-CoV-2 and are at risk of developing severe illness. The manufacturer, pharmaceutical and biotechnology giant Pfizer, developed it to patients avoid severe illness which can lead to hospitalization and death. It is meant to be administered at the first sign of infection.

The SARS-CoV-2 virus is spread through human-to-human transmission. Coughing and sneezing produces small droplets, which can be breathed in by someone else who then becomes infected. The droplets can also be passed on via hand contact, for example if someone touches their nose or face and then shakes hands.

Molnupiravir is an orally administered antiviral medication that inhibits the replication of certain RNA viruses, and is used to treat COVID-19.

Mari Carmen is the older sister of patient Juan Manuel, she takes care of her brother along with his wife. He was diagnosed with ALS in February 2020 and since then he has followed a clinical trial that includes Ketas (Ibudilast) which is not yet approved in Spain, where Juan Manuel lives.

Non-Hodgkin lymphoma (also called non-Hodgkin's lymphoma, NHL, or sometimes just lymphoma) is a type of cancer that starts in white blood cells known as lymphocytes. These cells are part of the body's immune system and they help the body fight infections.

Mislav was diagnosed with Grade II astrocytoma, a type of brain tumour, in 2018 and has struggled with the Croatian healthcare system to obtain a treatment, Fycompa, which was not approved in Croatia. TheSocialMedwork was able to help him access the medicine and he is now one of the patients that we are happy to not have anymore: the hospital where he is being treated has approved the import and treatment of Fycompa to him for free.

Bronchial asthma (also known as asthma) is a pulmonary disease, in which the airways narrow, swell and produce more mucus than necessary.

Iron deficiency is a state where the body lacks iron. It is the most common nutritional disorder in the world.

Multiple myeloma is a type of bone marrow cancer. Bone marrow is the soft tissue inside bones that produces the body's blood cells, including a type of white blood cell called plasma cell. Plasma cells play an important role in the immune system: they produce antibodies that help the body fight off germs.

Neuroblastoma is a type of cancer that is formed by nerve cells called neuroblasts. Usually, these very early forms of nerve cells develop into adult nerve cells, but in neuroblastoma these cells develop into cancer cells instead. This type of cancer occurs most commonly in infants and young children. Neuroblastoma normally forms in the fetus, before a child is born.

Bladder cancer - or urothelial cancer - most often begins in the cells that line the inside of the bladder, called urothelial cells. Urothelial cells are also found in other parts of the urinary system, such as the kidneys and the ureters. Urothelial cancer is found much more often in the bladder than in the other parts. But because people sometimes have tumors in these places, too, all of the urinary tract needs to be checked for tumors.

Spinal Muscular Atrophy (SMA) is an inherited disorder that affects nerves and muscles, which causes muscles to become increasingly weak. The cause is a shortage of the protein SMN, or ‘survival of motor neuron’. This protein is crucial for the function of nerves that control muscle activity. Without this protein, the motor neuron cells shrink and eventually die. This leads to muscle weakness seen in SMA..

Psoriatic arthritis is a type of arthritis that can occur in people who have the skin condition psoriasis. It is a chronic inflammatory disease that causes the joints to become swollen, stiff and painful. Approximately 30 percent of people with psoriasis develop psoriatic arthritis.

Prostate cancer is the most common cancer among men (after skin cancer). The prostate is a gland that is only found in men.

Thyroid cancer is a type of cancer that starts in the thyroid gland. The thyroid gland is located in front of the neck and is shaped like a butterfly.

Stomach cancer, or gastric cancer, begins when cells in the stomach start to grow out of control. These cells can grow into a tumor. Stomach cancer usually develops slowly, over the course of years

Skin cancer is the most common form of cancer. It forms when cells in the epidermis, the outermost layer of the skin, start to grow out of control.

Aduhelm (aducanumab) is a monthly intravenous infusion intended to slow cognitive decline by reducing amyloid beta plaques in the brain.

Ubrelvy (ubrogepant) is a calcitonin gene-related peptide (CGRP) receptor antagonist indicated for the acute treatment of migraine headaches with or without aura.

Tukysa (tucatinib) is a medication used in combination with trastuzumab and capecitabine for the treatment of adult patients with advanced, unresectable or metastatic HER2-positive breast cancer.

Radicut (edaravone) and Radicava (edaravone) is a medication for the treatment of patients with amyotrophic lateral sclerosis (ALS) and acute ischaemic stroke.

Trodelvy (sacituzumab govitecan-hziy) is an immune targeted therapy used for the 3rd line treatment of patients with metastatic triple-negative breast cancer (mTNBC).

Tagrisso (osimertinib) is a kinase inhibitor (targeted therapy) medication.

Vyndaqel (tafamidis) is a medicine indicated for the treatment of transthyretin amyloidosis in patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.

Tibsovo (ivosidenib) is an IDH1 inhibitor (targeted therapy) medication.

Enhertu (fam-trastuzumab deruxtecan-nxki) is an antibody-drug conjugate targeted therapy.

Chronic Lymphocytic Leukemia (CLL) is a slow-growing disease in which too many immature lymphocytes (white blood cells, which are cells of the immune system of the body) are found mostly in the blood and bone marrow.

Muscular Dystrophy (MD) refers to a group of over 30 inherited genetic and progressive diseases caused by a mutation in a person’s genes. The different types of MD vary in signs, symptoms and affect specific muscle groups.

Acute myeloid leukemia (AML) also known as acute myelocytic leukemia, acute myelogenous leukemia, acute granulocytic leukemia, and acute non-lymphocytic leukemia.