Sidra Medicine Establishes Gene Therapy Centre For Rare Paediatric Diseases
Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...
TheHealthSite10d
Raising Awareness On Spinal Muscular Atrophy: The Silent Battle Every Indian Should Know
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PMLiVE19h
Biogen’s higher dose spinal muscular atrophy regimen accepted for review by EMA/FDA
Biogen has announced that the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have started ...
Qatar Tribune on MSN1d
Qatar: Sidra establishes Gene Therapy Center for rare genetic diseases
The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...
People with spinal muscular atrophy demand urgent action from the Centre to reduce medicine costs
Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...
Pharm Exec5d
Biogen Seeks FDA Approval for a Higher Dosing Regimen of Spinraza for Spinal Muscular Atrophy
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
Healio27d
Gene therapy slows progression of spinal muscular atrophy type 2
Back to Healio Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease ...
The Week5d
Spinal muscular atrophy: Patients, activists seek health ministry’s intervention to bring down medicine cost
The health activists argue that the government is empowered with effective law and policy tools and therefore should ...
Cherai residents rallying to support toddler’s battle against rare disease
KOCHI: Residents of Cherai are rallying to support two-and-a-half-year-old Adharv, who is suffering from Spinal Muscular ...