B.C. family nearing finish in race to raise $2.8 million to treat baby's rare disease

8 August 2020 - Parents hope funds will pay for expensive therapy not approved in Canada for type 1 spinal muscular ...

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'I need something to move forward,' says Edmonton mom waiting for approval for son's life saving treatment

24 July 2020 - The family of a little boy in need of a $3 million treatment says they have ...

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Sarepta Therapeutics receives fast track designation for SRP-9001 micro-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy

24 July 2020 - Sarepta Therapeutics today announced that the U.S. FDA has granted fast track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin).  ...

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Gene therapy shows promise for haemophilia, but could be most expensive U.S. drug ever

20 June 2020 - Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with ...

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'Heartbreaking': Alberta families describe waiting for life-changing drug to be made available in Canada

17 July 2020 - More families are hoping a one-time treatment option for a rare condition will be made available ...

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Clock ticking on Edmonton family's hope for $2.8 million drug treatment for toddler son

 3 July 2020 - Family hoping Alberta Health will pay for new drug that must be administered by mid-July. ...

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Life-changing drug remains unfunded

1 July 2020 - Knowing his body is slowly deteriorating is ‘‘the scariest feeling ever’’. ...

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Germany first in EU to get Novartis’ SMA gene therapy, costing almost 2 million euros

24 June 2020 - Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal ...

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Teen with spinal muscular atrophy campaigns for wonder drug to survive

15 June 2020 - Taylor Fincham is one of 35 Kiwi children who suffer from spinal muscular atrophy. ...

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Cell and gene therapies: FDA official on COVID-19 impact

10 June 2020 - Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US ...

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Hawke's Bay mum's heartbreak as PHARMAC refuses spinal muscular atrophy drug for her two kids

9 June 2020 - Skylah-Rose Shaw 8 and her brother Zayden Shaw 10 suffer from spinal muscular atrophy a disease that ...

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Alnylam announces U.S. FDA has granted priority review of the lumasiran new drug application for the treatment of primary hyperoxaluria type 1

26 May 2020 - PDUFA date set for 3 December 2020. ...

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Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis

21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...

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B.C. teen with rare disease wins fight to receive costly drug from province

19 May 2020 - Health ministry won't explain change of heart in granting Miles Ambridge treatment for spinal muscular atrophy. ...

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AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy

19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...

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