Every year since 2008, Rare Disease Day has been held on the last day of February to raise awareness about the research that ...
Thirty-nine-year Bryan Samples is sharing his journey with SMA also known as Spinal Muscular Atrophy. It’s a rare disease ...
SMA patients and advocacy groups have called for urgent action to make life-saving treatments more accessible. The cost of ...
The mother took the drug daily for six weeks while the baby herself was given the drug orally, roughly one week after the ...
Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...
In her writ petition, Seba called for the continuation of her treatment with risdiplam beyond the Rs 50 lakh cap under the ...
On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...
The parents were both known carriers of SMN1 gene mutations that raised the risk of SMA, and sadly had previously had a child born with the disease who died at 16 months of age. Genetic testing of ...
More than 2 years later, the child shows no features of the disease. The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...
The ministry of health will provide services to patients who are suffering from hard-to-treat and rare diseases during the ...