Science Daily

New target found for treatment of spinal muscular atrophy

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...
The American Journal of Managed Care

Innovations in SMA Treatment: The Last Decade and Beyond

The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research. A ...
The American Journal of Managed Care

Other Investigational Treatment Options for SMA

Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, ...
Yahoo

Novartis wins approval to use SMA gene therapy in older patients

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. The Food and Drug Administration on Monday approved a ...
Devdiscourse

Kerala Expands Free Risdiplam Supply to SMA Patients Up to 25

Kerala's Health Department extends free Risdiplam supply to Spinal Muscular Atrophy patients up to 25 years, broadening the previous limit of 12 years. This initiative under the CARE program, launched ...
CNN

New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA® (nusinersen) Continue to Demonstrate Sustained Benefit From Treatment

After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...
MarketWatch

Novartis receives FDA approval for Itvisma(R), the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

The MarketWatch News Department was not involved in the creation of this content. -- Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients ...
MedPage Today

First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...
Business Wire

Five-year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of Spinal Muscular Atrophy (SMA) Achieved or Maintained the Ability to Sit ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...
Science Daily

'Prelude' to neuromuscular disease SMA may offer chances for better treatment

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...
Business Wire

Majority of Newborn Babies With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Able to Sit Independently After 1 Year of Treatment

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...
Newsweek

SMA Type 1: What to Know About Disorder Affecting Jesy Nelson’s Twins

Girlband star Jesy Nelson revealed in an emotional Instagram video that both of her twins had been diagnosed with a genetic condition called Spinal Muscular Atrophy (SMA), type 1. U.K.-based Nelson, ...