Baby Pia: almost 1 million Belgians pay for life-saving drug

18 September 2019 - More than 950,000 Belgians have responded to a couple seeking €1.9m ($2m; £1.7m) to cover the ...

Read more →

Three-year-old Matilda's ability to walk is giving hope to those with spinal muscular atrophy

18 September 2019 - The disease, which has been described as motor neurone disease for children, is the leading cause of ...

Read more →

The startup that manipulated data to get a miracle drug to market

14 September 2019 - AveXis had a revolutionary gene therapy for babies with a deadly disease, and sold to Novartis for ...

Read more →

Reeling from scandal, Novartis vows to voluntarily release info on manipulated data in the future

9 September 2019 - Seeking to appease angry regulators, Novartis committed to notifying the FDA within five business days of ...

Read more →

Kiwi toddler fights spinal muscular atrophy in Australia after PHARMAC stall access to life-saving drug

7 September 2019 - For the first time in her life, two-year-old Charlotte Bond is showing signs that one day ...

Read more →

Insurers pitch new ways to pay for million dollar therapies

5 September 2019 - Cigna and CVS Health are developing new programs to help employers afford gene therapies that can cost ...

Read more →

Cigna rolls out new plan to fully cover multi-million dollar gene therapies

5 September 2019 - Health insurer Cigna said on Thursday it had introduced a plan to fully cover costs for ...

Read more →

Magenta Therapeutics announces FDA regenerative medicine advanced therapy designation granted to MGTA-456 for the treatment of inherited metabolic disorders

4 September 2019 - Magenta Therapeutics today announced that the U.S. FDA granted regenerative medicine advanced therapy designation for MGTA-456, ...

Read more →

NHS funds eye gene therapy, after Novartis cuts £613,000 price

4 September 2019 - NICE has recommended the NHS should fund Novartis’ Luxturna for a rare eye disorder, after the ...

Read more →

NICE recommends novel gene therapy treatment for rare inherited eye disorder

4 September 2019 - It's estimated that 86 people would be eligible for treatment with voretigene neparvovec in England. ...

Read more →

Russia approves Spinraza to treat all SMA patients

29 August 2019 - Russia has become the first of the 15 former Soviet republics to approve Biogen’s Spinraza (nusinersen) ...

Read more →

An FDA report reveals the agency believed Novartis data problems were resolved months ago

27 August 2019 - Shortly before serious data problems surfaced in mid-March at a troubled Novartis unit, a Food and ...

Read more →

Life changing drugs almost nobody can afford

23 August 2019 - Patients suffering from rare diseases who had little hope for a treatment now have access to ...

Read more →

FDA focuses on Novartis delay in reporting drug-test data manipulation

22 August 2019 - Regulators zeroing in on the company’s two-month delay in launching a formal inquiry. ...

Read more →

FDA on Novartis data manipulation controversy: ‘we happened to be lucky’

21 August 2019 - Since the FDA blasted Novartis earlier this month over data manipulation, one of the major questions ...

Read more →