News Related to Spinal Muscular Atrophy

India pushes rare disease care with Rs 50 lakh support and focus on local innovation

May 05, 2026

India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.

Novartis’ Itvisma moves closer to EU approval for broader SMA population

Apr 24, 2026

Novartis received a positive CHMP opinion for Itvisma in spinal muscular atrophy patients aged 2 years and older. A European Commission decision is expected within about two months.

Novartis pipeline and growth targets remain central to investor focus

Apr 18, 2026

Novartis is emphasizing innovative medicines across oncology, immunology, neuroscience and gene therapy. The company targets mid-single-digit sales growth through 2027 and core operating income margins above 35%.

European Commission Approves First Treatment for Rare Mitochondrial Disease TK2d

Mar 31, 2026

The European Commission has approved KYGEVVI (doxecitine and doxribtimine) as the first treatment for thymidine kinase 2 deficiency (TK2d), an ultra-rare mitochondrial disease. Clinical studies showed the drug reduces death risk by 95% and helps 84% of patients regain motor functions. The approval covers pediatric and adult patients with symptom onset before age 12.

Zebrafish Model Prevents Unnecessary SMA Treatment After False-Positive Newborn Screening

Mar 21, 2026

Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.

Rare Disease Therapies Advance for Dravet Syndrome and Spinal Muscular Atrophy

Mar 05, 2026

New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.

Scholar Rock Prepares Apitegromab BLA Resubmission Following FDA Facility Reinspection

Mar 04, 2026

Scholar Rock plans to resubmit its biologics license application for apitegromab in spinal muscular atrophy following FDA reinspection of the Catalent Indiana fill-finish facility, with a U.S. launch anticipated in 2026.

AI Transforms Rare Disease Diagnosis as Awareness Day Highlights Patient Challenges

Mar 01, 2026

Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.

Rare Disease Day Highlights Diagnostic Delays and Access Barriers to Gene Therapy

Feb 28, 2026

International Rare Disease Day on February 28 underscores challenges in diagnosing and treating rare genetic conditions, with patients facing years-long diagnostic delays and geographic barriers to accessing gene therapies.

RNA-Targeting Small Molecules Gain Momentum as Drug Discovery Frontier

Feb 27, 2026

Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.