A Kerala government hospital distributed generic SMA medicine bearing a clinical trial label. The switch has raised concerns about regulatory approval, bioequivalence and parental consent. The issue is particularly sensitive because SMA is a rare genetic disorder that progressively weakens muscles and can be fatal in its more severe forms. Treatment can improve survival and slow disease progression, but the cost of medicines has historically put them beyond the reach of many Indian families. Kerala has been an exception. Since 2022, it has been the only Indian state to provide free SMA therapy to eligible children, and currently around 200 patients are estimated to benefit from the programme. It was expanded into the Kerala United Against Rare Diseases (KARE) programme in 2023, providing approved medicines and specialised care to children with SMA under the age of 12. The programme uses a hub-and-spoke model to coordinate treatment and has established a patient registry to help monitor beneficiaries. The generic had the potential to substantially reduce the cost of treatment and make public funding programmes more sustainable. This week, Sree Avittom Thirunal (SAT) Hospital in Thiruvananthapuram started distributing generic risdiplam. Parents of the patients, however, noticed that the packaging carried the words “Physician Sample” and “For Clinical Trials Only”, prompting fears that their children were being given an experimental drug instead of the medicine they had been receiving. It modifies the processing of SMN2 messenger RNA, helping the body produce more survival motor neuron (SMN) protein. A deficiency of this protein causes the progressive muscle weakness associated with the disease. Treatment is most effective when started early, particularly in infancy, but older patients can also benefit.

Original title: “Kerala SMA treatment row: Natsmart's clinical trial label raises concerns - India Today”

The Department of Health's adoption of affordable generic Risdiplam for Spinal Muscular Atrophy marks a major victory for patient access and public health Spinal Muscular Atrophy (SMA) is a rare, life-threatening genetic disorder that progressively weakens muscles, severely impairing both mobility and respiratory function. Although a complete cure remains elusive, Risdiplam is currently available as a pharmaceutical intervention capable of significantly decelerating disease progression. Timely administration of this medication is critical for enhancing patients’ quality of life and longevity. Roche’s branded therapeutic equivalent, Evrysdi (Risdiplam), costs about ₹6.2 lakh per bottle. Conversely, Natco’s generic version costs only ₹15,900 per bottle. Since a single bottle typically lasts 12 days, Natco’s monthly expenditure is about ₹30,000 (though actual costs may vary based on the patient’s age, dosage, and volume used). The availability of scientifically validated, universally recognised generic pharmaceuticals at lower cost is a major benefit for SMA patients and their families. Consequently, the Department of Health must disregard these unfounded media campaigns and steadfastly advance this decisive, patient-centric initiative. Dr B Ekbal is a former professor of neurosurgery and a public health activist. When authorised enterprises such as Natco introduce generic formulations, they must legally and scientifically demonstrate bioequivalence, proving that the drug enters the bloodstream in a manner comparable to the branded original. Under the Drugs and Cosmetics Act, regulatory approval for any generic pharmaceutical is granted only after successful completion of rigorous bioequivalence studies.

Original title: “Why recognising a generic drug for Spinal Muscular Atrophy is significant - The Week”

lrp12 cgg repeat expansions in patients with adult onset non 5q spinal muscular atrophy

Original title: “LRP12 CGG Repeat Expansions in Patients With Adult-Onset Non-5q Spinal Muscular Atrophy.”

consensus for the diagnosis and treatment of patients with spinal muscular atrophy sma in latin america

Original title: “Consensus for the Diagnosis and Treatment of Patients With Spinal Muscular Atrophy (SMA) in Latin America.”

Projected by some analysts as a future blockbuster, Isembyld is the first “muscle-targeted” treatment for the disease and, in clinical testing, helped improve motor function in people on standard therapies.

Original title: “Scholar Rock SMA drug approved by FDA”

frequency and circumstances of falls events in people living with spinal and bulbar muscular atrophy a cross sectional survey

Original title: “Frequency and Circumstances of Falls Events in People Living With Spinal and Bulbar Muscular Atrophy: A Cross-Sectional Survey.”

integrating lna qpcr and full length smn1 sequencing for precision sma carrier screening large scale validation in 30 849 individuals

Original title: “Integrating LNA-qPCR and full-length SMN1 sequencing for precision SMA carrier screening: large-scale validation in 30,849 individuals.”

spinal muscular atrophy in adult neurology services in india

Original title: “Spinal Muscular Atrophy in Adult Neurology Services in India.”

muscle mri as an imaging biomarker of muscle damage in patients with spinal and bulbar muscular atrophy

Original title: “Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.”

spinal muscular atrophy all babies in england to be screened for rare neuromuscular disease

Original title: “Spinal muscular atrophy: all babies in England to be screened for rare neuromuscular disease.”

The Food and Drug Administration (FDA) is classifying the Spinal Muscular Atrophy newborn screening test system into class II (special controls). The special controls that apply to the device type are identified in this order and will be part of the codified language for classification of the Spinal Muscular Atrophy newborn screening test system. We are taking this action because we have determined that classifying the device into class II will provide a reasonable assurance of safety and effect

Original title: “Medical Devices; Immunology and Microbiology Devices; Classification of the Spinal Muscular Atrophy Newborn Screening Test System”

cost effectiveness of universal newborn screening and disease modifying therapies for spinal muscular atrophy in taiwan

Original title: “Cost-Effectiveness of Universal Newborn Screening and Disease-Modifying Therapies for Spinal Muscular Atrophy in Taiwan.”

After trial flops in spinal muscular atrophy, depression and bipolar disorder—and a costly rare disease drug rejection—Biohaven is undergoing a reset, recasting its former SMA candidate for obesity.

Original title: “Biohaven looks to obesity to bounce back from run of clinical and regulatory failures”

advancements in prenatal diagnosis and potential fetal therapies for spinal muscular atrophy

Original title: “Advancements in Prenatal Diagnosis and Potential Fetal Therapies for Spinal Muscular Atrophy.”