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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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14th Annual RNA Therapeutics Conference

pharmaphorum

Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Heads of Translational Medicine. Website: [link].

RNA 52
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New England Journal of Medicine Publishes 12-Week Results from Study.

The Pharma Data

AbbVie (NYSE: ABBV) today announced that the New England Journal of Medicine ( NEJM ) has published 12-week results from the Phase 3 ADVANCE trial evaluating atogepant for the preventive treatment of migraine in adults who meet criteria for episodic migraine. The full NEJM article is available at nejm.org.

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Artificial intelligence could be new blueprint for precision drug discovery

The Pharma Data

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. “Academic labs and pharmaceutical and? the success rates in drug discovery?are

Drugs 52
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Latest analyses of Vitrakvi (larotrectinib) reaffirm powerful efficacy and tolerability profile for both adult and pediatric patients with TRK fusion cancer

The Pharma Data

Data from four distinct analyses for Vitrakvi (larotrectinib) showcase its sustained clinical benefit for patients with solid tumors harboring an NTRK gene fusion (also referred to as TRK fusion cancer). About TRK Fusion Cancer TRK fusion cancer occurs when an NTRK gene fuses with another unrelated gene, producing a chimeric TRK protein.

Gene 52
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positive result of phase III study with radioligand therapy 177Lu-PSMA-617 in patients with advanced prostate cancer

The Pharma Data

These groundbreaking data confirm our belief in the potential of 177 Lu-PSMA-617 to reimagine outcomes for these patients through phenotypic precision medicine. We would not be able to realize our commitment to reimagining medicine without the partnership of patients and their families.”. About Advanced Prostate Cancer.

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Hansa Biopharma provides business update incl. certain key financials ahead of the JP Morgan Global Healthcare Conference

The Pharma Data

Regulatory discussions with the European Medicines Agency (EMA) and FDA will be initiated to determine the regulatory path forward for imlifidase in anti-GBM. Imlifidase as pre-treatment ahead of gene therapy in DMD and LGMD. Imlifidase as pre-treatment ahead of gene therapy in DMD and LGMD.