Rare Disease Day 2024 - Neuraxpharm & Minoryx

#RareDiseaseDay 2024 is a day that is focused on raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and carers.
At Neuraxpharm we help patients suffering from a range of rare diseases, but this year, we want to focus on one in particular: Cerebral Adrenoleukodystrophy - cALD.
cALD is a terrible disease that causes demyelinating brain lesions which can become rapidly progressive, leading to acute neurological decline and death in three to four years and for which there is currently no pharmacological treatment available. You can find out more here: www.neuraxphar...
We have produced a special film that we have made with our partners Minoryx, to raise awareness of this disease as it is critical that symptoms are identified as early as possible. The film features world renowned expert Prof Fanny Mochel, the Director of the Reference Center for Leukodystrophies - La Pitié-Salpêtrière Hospital, Paris, on the disease and the need for new treatments, and Karen Harrison, Support Services Manager of Alex, the Leukodystrophy Charity, who speaks movingly about the patient experience.
Watch the film and please spread the word.