• Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases
  • IgA nephropathy (IgAN), while rare, is the most common form of glomerulonephritis, affecting mostly young adults with no approved treatment option and significant risk to progress to e
  • Huntington’s disease is a rare, inherited neurodegenerative disease that leads to progressive disability and death

  • There are no approved disease modifying therapies that delay disease onset or slow progression of the disease

  • Branaplam (LMI070) is an orally administered, small molecule RNA splicing modulator that could potentially reduce the levels of mutant huntingtin protein

Basel, October …

  • If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients with hypercholesterolemia or mixed dyslipidemia1
  • Cardiovascular disease (CVD) claims 3.9 million lives annually in Europe2, and 80% of high-risk patients do not reach guideline-recommended low-density lipoprotein cholesterol (LDL-C) targets despite the widespread …
  • The European Medicines Agency has granted iptacopan a priority medicines (PRIME) designation in C3 glomerulopathy (C3G). 
  • PRIME is granted for medicines that may offer major therapeutic advance or benefit patients without treatment options.
  • C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need.1

2.2 billion people are blind or visually impaired worldwide. Half of them lack access to eye care. Together, we are working to change that. This World Sight Day, Novartis is proud to partner with the International Agency for the Prevention of Blindness (IAPB) to expand equitable access to eye care in remote communities with the initiation of early detection eye health screening programs.

In Rwanda, it is estimated that more than 65,000 people (0.6% of the population) are blind in both eyes and that 12% of the population have a correctable refractive error or blurred vision, requiring corrective lenses.1

In this country of more than 12 million residents, there are only 18 ophthalmologists.

As one of those ophthalmologists, and the only one within a catchment area of 1.2 million people from Rwanda and nearby countries, Dr. Theophile Tuyisabe has an enormous responsibility. Each day he commutes an hour to the Eye Unit at the Kabgayi Hospital, treats …

  • In a post-hoc analysis of HAWK and HARRIER, fewer Beovu (brolucizumab) patients had early persistent fluid (12.5% vs. 20.4% of aflibercept patients), defined as the presence of intra-retinal fluid and/or sub-retinal fluid through week 12 of treatment1
  • Patients with early persistent fluid treated with Beovu experienced greater gains in best-corrected visual acuity (BCVA) at week 96 …
  • Nearly two-thirds of patients (65.6%) in STR1VE-EU have already achieved developmental motor milestones not observed in the natural history of SMA Type 1 at a mean duration of follow-up of 10.6 months, including patients with a more severe phenotype compared to previous studies
  • Two-thirds of patients (66.7%) were free of feeding support, an important indicator of stabilization/halting of disease progression
  • New interim Phase 3 STR1VE-EU data presented at WMS support the robust clinical evidence that …

COVID-19’s existence anywhere poses a threat to communities everywhere. The health, social, and economic impacts can only be addressed through the collective actions of stakeholders across private, public, and philanthropic sectors in partnership with civil society. As organizations dedicated to improving and protecting global health, with our varied skills, roles, and resources, we remain committed to doing our part in ending this pandemic worldwide. Earlier this year AstraZeneca; Bayer; bioMérieux; Boehringer Ingelheim; Bristol Myers Squibb; Eisai; Eli Lilly; Gilead; GSK; Johnson & …

For the past six decades, Novartis has been working to advance heart health around the world, from transforming the standard of care for cardiovascular conditions to strengthening healthcare systems in lower income communities.

Cardiovascular disease (CVD) is a general term for conditions affecting the heart or blood vessels. Although average CVD mortality rates have fallen by 61% over the 50-year period since 19601, CVD remains the number one killer worldwide, causing one out of three deaths globally2.

CVD has many causes: from smoking, diabetes, high …