The Snail spirit animal stands for sensuality and fluidity. The Snail symbolism reminds us that we should have diversity in our lives. To have the Snail as a totem animal, it means that one can be quite shy and reserved with people they don't know well. SMA impacts approximately one in 11,000 live births in the U.S., [v] is a leading cause of genetic death among infants 3and causes a range of disability in teenagers and adults. 4 Traditionally, treatments are reimbursed on a (sub-)population level. However, the importance of patient-centeredness as a key value is becoming increasingly apparent. Importantly, as scientific advances permit greater personalisation when treating complex diseases, it is critical that value assessment frameworks strike a balance between decision making at population and individual levels [ 1]. This individual-focussed and more precise approach is especially important in rare diseases. Often, the narrow eligibility criteria in clinical trials lead to a homogeneous study population, but at the same time exclude many individuals. Only relying on data collected under such strict inclusion conditions inevitable leads to a knowledge gap, and consequently to a situation in which individuals are eventually left behind without the prospect of any treatment. Early and continuous involvement of patient experts helps to proactively identify such challenges on the one hand, and jointly find the best possible solutions for the patient community on the other. Nestler-Parr S, Korchagina D, Toumi M, Pashos CL, Blanchette C, Molsen E, Morel T, Simoens S, Kalo Z, Gatermann R, Redekop W. Challenges in research and health technology assessment of rare disease technologies: report of the ISPOR rare disease specialist interest group. Value Health. 2018. https://doi.org/10.1016/j.jval.2018.03.004. Life Lesson & Challenge: As the first vowel of their name is 'E', people named Mejat are given the life lesson of experiencing a better reaction to personal stress, whilst the life challenge is linked to refraining from the pursuit of an unrealistic social image.

Perfetto EM, Oehrlein EM, Boutin M, Reid S, Gascho E. Value to whom? The patient voice in the value discussion. Value Health. 2017. https://doi.org/10.1016/j.jval.2016.11.014. Alexandre was previously member of the Board of Directors of the Institute for Stem cells research (I Stem, Evry, France. From 2005 to 2017) and AFM-Telethon (Evry, France. From 2010 to 2017). He is now implicated in several international networks and consortia such as the European NeuroMuscular Center (ENMC), the ERN-EuroNMD, European Joint Program on Rare Diseases (EJP-RD) and the International Rare Diseases Research Consortium (IRDiRC).Swift B, Jain L, White C, Chandrasekaran V, Bhandari A, Huges DA, Jadhav PR. Innovation at the intersection of clinical trials and real-world data science to advance patient care. Clin Transl Sci. 2018. https://doi.org/10.1111/cts.12559.

Anna Arellanesová, Elizabeth Vroom, Simona Bellagambi, Birthe Byskov Holm, Alexandre Méjat, Rebecca Tvedt Skarberg and Kirsten Johnson also continue to serve on our Board of Directors. Bohannon L, Franklin E. Value Assessment that puts patients at the forefront. Value Outcomes Spotlight. 2021;7(1):29–31.Bione S, Maestrini E, Rivella S, Mancini M, Regis S, Romeo G, et al. Identification of a novel X-linked gene responsible for Emery-Dreifuss muscular dystrophy. Nat Genet. 1994;8(4):323–327. doi: 10.1038/ng1294-323. The initiative includes an interactive map and policy recommendations on access to care options for people living with SMA Laboratoire de Cardiogénétique Moléculaire, Centre de Biologie et Pathologie Est, Hospices Civils de Lyon, Lyon, France. Account icon An icon in the shape of a person's head and shoulders. It often indicates a user profile. From Malay meja, from Portuguese mesa ( “ table ” ), from Old Galician-Portuguese, from Latin mēnsa.