spinal muscular atrophy

Zolgensma

Scholar Rock SMA drug shows muscle function benefit

Clinical Trials Arena

Mon, 03/17/25 - 10:59 am

Scholar Rock

apitegromab

Novartis’ Phase III spinal muscular atrophy therapy trial meets primary endpoint

Clinical Trials Arena

Thu, 01/2/25 - 11:25 am

OAV101 IT

Biohaven fails phase 3 SMA test but plans talks with FDA, plots move into obesity

Fierce Biotech

Mon, 11/25/24 - 11:22 am

Biohaven

taldefgrobep alfa

FDA

obesity

Genentech’s Evrysdi helped babies with SMA reach rare milestones in Phase II trial

Clinical Trials Arena

Tue, 10/15/24 - 10:16 am

Roche

Evrysdi

Biogen fills in some of the blanks on its trial of a high-dose version of Spinraza

Fierce Pharma

Wed, 10/9/24 - 09:52 am

Scholar Rock scores with ‘surprise’ success in SMA drug study

BioPharma Dive

Mon, 10/7/24 - 11:52 am

Scholar Rock

apitegromab

obesity

Biogen to seek approval for high-dose version of SMA stalwart Spinraza after trial win

Fierce Pharma

Wed, 09/4/24 - 10:49 pm

Spinal muscular atrophy market to reach $3bn across 7MM by 2033

Clinical Trials Arena

Wed, 07/17/24 - 09:12 pm

PTC to sell spinal muscular atrophy drug royalties for $1B

BioPharma Dive

Thu, 10/19/23 - 11:34 am

PTC Therapeutics

Evrysdi

Royalty Pharma

Spinal Muscular Atrophy: Four trials to watch over the next 12 months

Clinical Trials Arena

Fri, 08/11/23 - 11:39 am

Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk

BioSpace

Mon, 07/3/23 - 11:38 am

Genentech

Evrysdi

Novartis sales of Zolgensma gene therapy slow as market shifts

BioPharma Dive

Tue, 10/25/22 - 11:02 pm

Zolgensma

Novartis reports deaths of two patients treated with Zolgensma gene therapy

BioPharma Dive

Thu, 08/11/22 - 11:32 pm

Scholar Rock in a hard place as biotech lays off 25% of staff, narrows pipeline and loses CMO

Fierce Biotech

Tue, 05/17/22 - 10:45 am

Scholar Rock

layoffs

New Three-Year Data for Genentech’s Evrysdi (risdiplam) Show Long-Term Improvements in Survival and Motor Milestones in Babies With Type 1 Spinal Muscular Atrophy (SMA)

BioSpace

Fri, 04/29/22 - 10:30 am

Biogen Announces New Spinraza Data Ahead of Presentation

BioSpace

Mon, 03/14/22 - 11:39 pm

New Data Reinforces Impact of Novartis' Gene Therapy on SMA

BioSpace

Mon, 03/14/22 - 10:46 am

Roche's aim to widen Evrysdi's label nabs speedy FDA review in young spinal muscular atrophy patients

Zolgensma

Fierce Pharma

Tue, 01/25/22 - 10:54 am

Biogen brings in a successor to Spinraza

BioPharma Dive

Tue, 01/4/22 - 10:49 pm

Ionis Pharmaceuticals

BIIB115

antisense