Scholar Rock Sets Sights on $2 Billion Apitegromab Revenue Amid Spinal Muscular Atrophy Market Growth
Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.
The American Journal of Managed Care1d
Phase 3 STEER Study of SMA Gene Therapy Meets Primary End Point
Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...
Advocates rally at State House against potential cuts to special education funding
Advocates rallied outside the State House on Tuesday for special education funding.The group urged lawmakers to allocate more ...
Hosted on MSN2d
Broader population could benefit from Novartis spinal muscular atrophy treatment
Positive results from its Phase III STEER study mean Novartis could boost sales of its OAV101 IT drug for spinal muscular ...
Winnipeg Free Press3d
‘I’m being forced into a place’
Crystal Rondeau’s muscle wasting disease has left her with only one finger to press an alarm button or send a text. The ...
I've Traveled to 44 Countries and All 7 Continents in a Wheelchair — Here's What It's Like
Accessible travel blog founder Cory Lee is committed to helping other wheelchair users travel the world freely.
MP asks government to authorise local manufacture of drug for rare disease to slash cost
A Rajya Sabha MP urges the health minister to enable local production of Risdiplam, a vital drug for spinal muscular atrophy, ...
Experts seek affordable access to medicines for treating Spinal Muscular Atrophy
Haris Beeran urges government intervention to make affordable SMA medicines available in India, addressing the high costs and patent issues.
TMCnet10d
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
Under this newly granted orphan drug designation, NMD670 is currently being evaluated in a Phase 2 clinical trial in adult patients with any genetically confirmed Type 1 or Type 2 CMT subtype NMD ...
Bloomberg L.P.17d
Novartis Says New Drug Helps Children With Rare Spinal Condition
Novartis AG said a new experimental medicine improved motor function in children with a form of spinal muscular atrophy, the devastating disorder targeted by its gene therapy Zolgensma ...
MedPage Today20d
Kids with Spinal Muscular Atrophy Rate Their Own Health
—A new study analyzed health-related quality of life (HRQoL) using 5 generic and neuromuscular disease-specific rating scales in German children with spinal muscular atrophy (SMA), most of whom ...
CBC.ca28d
A Manitoban is losing his ability to move his muscles. A drug might prevent that, but the province won't pay
He hasn't treated Bray, but has worked on a research study of other adults with Type 2 spinal muscular atrophy that showed positive effects from taking risdiplam. Around 90 per cent of his ...