A nine-year-old boy's life has been "fundamentally changed" thanks to approved treatments for spinal muscular atrophy (SMA), allowing him to reach significant milestones like starting mainstream school despite a grim prognosis. #SMA #RareDiseases #HealthcareInnovation #GB
A nine-year-old boy named Ezra, who has spinal muscular atrophy (SMA), has seen his life transformed by treatments recently approved for routine use in the NHS, allowing him to reach milestones that were once thought unattainable after a grim prognosis. #SpinalMuscularAtrophy #RareDiseases #HealthInnovation #GB
A nine-year-old boy named Ezra, who has spinal muscular atrophy, has seen a significant improvement in his life and the ability to start mainstream school following the NHS approval of nusinersen and risdiplam treatments, which were previously restricted but have now become widely available. #SpinalMuscularAtrophy #SMA #RareDisease #GB
Now, with the #BiotechAct, medical devices reform and the pharmaceutical package, we want to accelerate innovation and improve access to new diagnostics and therapies for rare and complex conditions. ➡️ Rare should never mean invisible. #RareDiseases #BiotechInnovation #HealthcareAccess #Nospecificcountrycodesarementionedinthetweet.
Rare diseases may each affect few patients, but together they impact millions of families across 🇪🇺. We must strengthen awareness, research & cooperation. European Reference Networks connect expertise across Member States. The EU has invested €3.2bn in rare disease research. ⤵️ #RareDiseases #HealthResearch #EURareDiseases #EU
In a significant milestone for the treatment of epidermolysis bullosa, two Andalusian patients, Leo and Adrián, have become the first in Spain to receive gene therapy with the drug Vyjuvek, promising to greatly improve their quality of life by reducing pain and healing time, although challenges remain regarding equitable access to this life-changing treatment across the country. #GeneTherapy #HealthEquity #RareDiseases #ES #ES #ES #ES
