Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi ™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support...
Source: Roche Media News - September 28, 2020 Category: Pharmaceuticals Source Type: news

Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi ™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support...
Source: Roche Investor Update - September 28, 2020 Category: Pharmaceuticals Source Type: news

SMA1 Infant Life-Saving Zolgensma Therapy Unavailable in Canada
Ground-breaking new drug Zolgensma that could stop progression of Spinal Muscular Atrophy Type 1 (SMA1) is the most expensive medication in the world (Source: Disabled World)
Source: Disabled World - September 15, 2020 Category: Disability Tags: Pharmaceutical Source Type: news

Can a rival to the world's most expensive drug help George with his muscle-wasting illness?
George Frost, 9, pictured with his sister Evie, left, was diagnosed with spinal muscular atrophy aged one. The youngster from Kent saw his muscles waste away until he was placed on a clinical trial. (Source: the Mail online | Health)
Source: the Mail online | Health - September 5, 2020 Category: Consumer Health News Source Type: news

Treatments for Inherited Neuromuscular Diseases of Childhood Treatments for Inherited Neuromuscular Diseases of Childhood
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 27, 2020 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

A multicenter look at gene therapy for spinal muscular atrophy
(Nationwide Children's Hospital) A new study confirms the safety and efficacy of gene therapy in children with spinal muscular atrophy under two years old. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - August 25, 2020 Category: Biology Source Type: news

FDA Approves First Oral Drug for Spinal Muscular Atrophy, Evrysdi (Risdiplam)
MONDAY, Aug. 10, 2020 -- The U.S. Food and Drug Administration has approved the first oral, at-home drug for spinal muscular atrophy (SMA), the agency announced Friday. Evrysdi (risdiplam) was approved to treat patients ages 2 months and older with... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - August 10, 2020 Category: General Medicine Source Type: news

FDA approves Roche ’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older
Roche announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi ™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. (Source: Roche Media News)
Source: Roche Media News - August 10, 2020 Category: Pharmaceuticals Source Type: news

FDA approves Roche ’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older
Roche announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi ™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. (Source: Roche Investor Update)
Source: Roche Investor Update - August 10, 2020 Category: Pharmaceuticals Source Type: news

FDA Approves Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older
South San Francisco, CA -- August 7, 2020 -- Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - August 8, 2020 Category: Drugs & Pharmacology Source Type: news

FDA OKs First Oral Treatment for Spinal Muscular Atrophy FDA OKs First Oral Treatment for Spinal Muscular Atrophy
The FDA has approved risdiplam (Evrysdi) as the first oral treatment for adults and children at least 2 months of age with spinal muscular atrophy.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 7, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

Nusinersen with screening for spinal muscular atrophy cost effective?
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - July 1, 2020 Category: Drugs & Pharmacology Source Type: news

Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Media News - June 12, 2020 Category: Pharmaceuticals Source Type: news

Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Investor Update - June 12, 2020 Category: Pharmaceuticals Source Type: news

Treat early or wait? Experts ponder best way to manage milder forms of spinal muscular atrophy
(IOS Press) The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. Now, in a report published in the Journal of Neuromuscular Diseases, German researchers argue for an earlier start of treatment to prevent permanent nerve damage, challenging recommendations originally proposed by a group of American experts that suggests a strict follow-up strategy for children expected to have less severe disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 11, 2020 Category: International Medicine & Public Health Source Type: news

New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the i...
Source: Roche Media News - May 22, 2020 Category: Pharmaceuticals Source Type: news

New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the i...
Source: Roche Investor Update - May 22, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p
Source: Roche Media News - April 28, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p
Source: Roche Investor Update - April 28, 2020 Category: Pharmaceuticals Source Type: news

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Brittany Stineman worries her  3-year-old son, Nash, won't survive Covid-19 if he catches it. He has a rare form of spinal muscular atrophy, and she is fighting to protect him. Watch more human stories of the pandemic. (Source: CNN.com - Health)
Source: CNN.com - Health - April 9, 2020 Category: Consumer Health News Source Type: news

U.S. regulators delay decision on Roche SMA drug risdiplam to Aug. 24
U.S. regulators delayed a decision on Roche's spinal muscular atrophy drug risdiplam, the Swiss drugmaker said on Tuesday, after the company submitted more data in February on the medicine against the genetic muscle-wasting disease. (Source: Reuters: Health)
Source: Reuters: Health - April 7, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Media News - April 7, 2020 Category: Pharmaceuticals Source Type: news

Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Investor Update - April 7, 2020 Category: Pharmaceuticals Source Type: news

How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients. 
  Now pharma needs to identify the ones that will move the dial commercially. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.”  To...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news

How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients. 
  Now pharma needs to identify the ones that will really move the dial. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.”  To stay ...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news

How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients. 
  Now pharma needs to identify the ones that will really move the dial. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.”  To stay ...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news

EMA Panel Backs Zolgensma Gene Therapy for SMA EMA Panel Backs Zolgensma Gene Therapy for SMA
Zolgensma is a gene therapy that addresses the genetic root cause of spinal muscular atrophy by replacing the defective or missing SMN1 gene to halt disease progression.International Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 28, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

Novartis wins key European recommendation for gene therapy Zolgensma
Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months. (Source: Reuters: Health)
Source: Reuters: Health - March 27, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

New Financing Could Help BillionToOne Expand Product Offerings
BillionToOne wants to expand its diagnostic offerings and its most recent financing could give it a push in that direction. The Menlo Park, CA-based company has raised $15 million in a follow-on to its series A round. With this financing, the company BillionToOne has brought in more than $32.5 million. The private company noted previous investors, Hummingbird Ventures and NeoTribe Ventures led the investment round with participation from Y Combinator, Libertus Capital, Pacific 8 Ventures, Civilization Ventures, 500 Startups Istanbul, and HOF Capital. BillionToOne said it will use the proceeds to support the commercial expa...
Source: MDDI - March 11, 2020 Category: Medical Devices Authors: Omar Ford Tags: IVD Business Source Type: news

Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 06 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)1 was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),2 a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for risdi...
Source: Roche Media News - February 6, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 6 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)[1]was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),[2] a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for ris...
Source: Roche Investor Update - February 6, 2020 Category: Pharmaceuticals Source Type: news

Reminder: Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
  (Source: Roche Investor Update)
Source: Roche Investor Update - January 31, 2020 Category: Pharmaceuticals Source Type: news

Roche's SMA drug trial win bolsters $2 billion sales prospects
Roche's spinal muscular atrophy (SMA) drug risdiplam hit another clinical trial target, the Swiss drugmaker said on Thursday, which analysts said bolsters its prospects of reaching $2 billion in annual sales. (Source: Reuters: Health)
Source: Reuters: Health - January 23, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
             Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was con...
Source: Roche Investor Update - January 23, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
             Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was con...
Source: Roche Media News - January 23, 2020 Category: Pharmaceuticals Source Type: news

Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
  (Source: Roche Investor Update)
Source: Roche Investor Update - January 17, 2020 Category: Pharmaceuticals Source Type: news

Roche aims to 'underwhelm' on SMA drug price to challenge rivals
Swiss drugmaker Roche plans to price its oral spinal muscular atrophy drug (SMA) risdiplam aggressively to challenge two of the world's most expensive medicines, Biogen's Spinraza and Novartis's gene therapy Zolgensma. (Source: Reuters: Health)
Source: Reuters: Health - January 14, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community. (Source: The Scientist)
Source: The Scientist - January 7, 2020 Category: Science Tags: News & Opinion Source Type: news

Spinraza funding recommended for spinal muscular atrophy in NZ
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - January 1, 2020 Category: Drugs & Pharmacology Source Type: news

Novartis in talks with patients upset about lottery-like gene therapy giveaway
Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease. (Source: Reuters: Health)
Source: Reuters: Health - December 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Novartis plans to give away world's costliest therapy to some patients
Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug program that one patient group worried was a "health lottery" that could neglect some babies. (Source: Reuters: Health)
Source: Reuters: Health - December 19, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
The animals lived longer and showed milder symptoms than untreated mice, although they didn't survive as long as wildtype mice. (Source: The Scientist)
Source: The Scientist - December 11, 2019 Category: Science Tags: News & Opinion Source Type: news

Novartis R & D boss says doesn't see big opportunity in oral SMA therapy
Novartis research head Jay Bradner said on Thursday the Swiss drugmaker is focusing drug development efforts against spinal muscular atrophy on gene therapy Zolgensma and retreating from oral therapy like its molecule LMI070, also called branaplam. (Source: Reuters: Health)
Source: Reuters: Health - December 5, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Novartis's $90 million Swiss factory to help solve cell therapy bottleneck
Swiss drugmaker Novartis's new $90 million cell and gene therapy factory in northern Switzerland is on track to begin commercial production of treatments, including Kymriah for cancer and Zolgensma for spinal muscular atrophy, in 2020. (Source: Reuters: Health)
Source: Reuters: Health - November 28, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. (Source: World Pharma News)
Source: World Pharma News - November 27, 2019 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

Roche eyes quick FDA approval in SMA race with Biogen, Novartis
Roche expects its risdiplam medicine for spinal muscular atrophy (SMA) to get U.S. approval by May, the Swiss drugmaker said on Monday, as it takes on Novartis and Biogen in the lucrative rare disease area. (Source: Reuters: Health)
Source: Reuters: Health - November 25, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. (Source: Roche Media News)
Source: Roche Media News - November 25, 2019 Category: Pharmaceuticals Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. (Source: Roche Investor Update)
Source: Roche Investor Update - November 25, 2019 Category: Pharmaceuticals Source Type: news

SUNFISH: Risdiplam Meets Primary Endpoint in SMA SUNFISH: Risdiplam Meets Primary Endpoint in SMA
Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in the pivotal part 2 of the SUNFISH trial.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 13, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

Roche closer to breaking into lucrative SMA market with positive trial results
Positive trial results for an oral drug to treat spinal muscular atrophy (SMA) are bringing Roche one step closer to breaking into the high-cost treatment area. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - November 11, 2019 Category: Pharmaceuticals Source Type: news